Toolkit/CRISPR/Cas9
CRISPR/Cas9
Also known as: Cas9, CRISPR, CRISPR-Cas9, CRISPR/Cas9, CRISPR-Cas9 genome editing technique, CRISPR-Cas9 technology, multiplex CRISPR/Cas9, Type II Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9 genome editing system
Taxonomy: Mechanism Branch / Architecture. Workflows sit above the mechanism and technique branches rather than replacing them.
Summary
CRISPR/Cas9 is a bacterial type II genome editing system repurposed as a programmable nuclease for target DNA cleavage and site-specific genome modification. The supplied evidence states that it was engineered for gene editing in mammalian cells by 2013 and is used to interrupt gene expression through cleavage of target DNA.
Usefulness & Problems
Why this is useful
The supplied literature describes CRISPR/Cas9 as accelerating gene editing applications and expanding gene therapy from semi-random gene addition to site-specific genome modification. It is also described as a potentially powerful and effective platform in cancer therapy contexts, reflecting utility where efficient and accurate genome modification is needed.
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By 2013, CRISPR/Cas9 systems had been engineered to allow gene editing in mammalian cells.
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With the advent of novel programmable nucleases, such as CRISPR/Cas9, it has been possible to expand the applications of gene therapy beyond semi-random gene addition to site-specific modification of the genome
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CRISPR-Cas9 can be employed to rapidly engineer immune cells and oncolytic viruses for cancer immunotherapeutic applications.
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CRISPR-Cas9 has shown an unprecedented clinical potential to discover novel targets for cancer therapy and to dissect chemical-genetic interactions, providing insight into how tumours respond to drug treatment.
Problem solved
This tool addresses the problem of making targeted genomic changes rather than relying on semi-random gene addition. The evidence also indicates that it can interrupt gene expression by cleaving target DNA, providing a programmable route to gene disruption.
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By 2013, CRISPR/Cas9 systems had been engineered to allow gene editing in mammalian cells.
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CRISPR-Cas9 can be employed to rapidly engineer immune cells and oncolytic viruses for cancer immunotherapeutic applications.
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CRISPR-Cas9 has shown an unprecedented clinical potential to discover novel targets for cancer therapy and to dissect chemical-genetic interactions, providing insight into how tumours respond to drug treatment.
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Because of its high efficiency and accuracy, the CRISPR-Cas9 genome editing technique has recently emerged as a potentially powerful tool in the arsenal of cancer therapy.
Problem links
addresses the need for targeted treatments against viral infection in KSHV-associated malignancies
LiteratureIt is positioned as a candidate virus-directed targeted treatment where current therapies remain suboptimal.
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It is positioned as a candidate virus-directed targeted treatment where current therapies remain suboptimal.
addressing limitations of CAR-T therapy in solid tumors
LiteratureIt is being explored to help enhance CAR-T cell specificity, improve resistance to immunosuppressive signals, and optimize in vivo functionality.
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It is being explored to help enhance CAR-T cell specificity, improve resistance to immunosuppressive signals, and optimize in vivo functionality.
editing genetic material as a therapeutic strategy
LiteratureIt offers a route to directly edit genetic material rather than only regulating expression.
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It offers a route to directly edit genetic material rather than only regulating expression.
editing mutated hemophilia genes
LiteratureIt addresses the goal of permanently correcting or compensating for F8 or F9 defects by precise editing or targeted cDNA integration.
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It addresses the goal of permanently correcting or compensating for F8 or F9 defects by precise editing or targeted cDNA integration.
enables controlled generation of arrhythmia models beyond patient-derived backgrounds
LiteratureIt helps build controlled arrhythmia models and supports targeted manipulation of ion-channel determinants of electrophysiology. This expands modeling beyond only naturally patient-derived variants.
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It helps build controlled arrhythmia models and supports targeted manipulation of ion-channel determinants of electrophysiology. This expands modeling beyond only naturally patient-derived variants.
enables introduction of disease-associated variants into flies for in vivo study
LiteratureIt helps convert human genetic findings into tractable fly models for functional study. This supports interpretation of newly discovered variants.
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It helps convert human genetic findings into tractable fly models for functional study. This supports interpretation of newly discovered variants.
enables targeted DNA sequence editing with guide-directed specificity
LiteratureIt enables targeted genome editing and gene-function studies in poultry, including knockout-based functional genomics and trait engineering. The paper also frames it as less contentious than transgenic integration-based methods when no exogenous DNA is integrated.
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It enables targeted genome editing and gene-function studies in poultry, including knockout-based functional genomics and trait engineering. The paper also frames it as less contentious than transgenic integration-based methods when no exogenous DNA is integrated.
enables targeted genetic manipulation of cells to improve tissue construct functionality
LiteratureIt helps make engineered cells and tissues more functional and physiologically relevant. The review frames it as a way to genetically tailor tissue constructs for regenerative medicine.
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It helps make engineered cells and tissues more functional and physiologically relevant. The review frames it as a way to genetically tailor tissue constructs for regenerative medicine.
enables targeted genetic modification of HSCs
LiteratureIt addresses the need for targeted genetic modification of HSCs to improve antitumor therapeutic potential.
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It addresses the need for targeted genetic modification of HSCs to improve antitumor therapeutic potential.
enables targeted genome modification in organoid systems
LiteratureIt helps enable precise modeling of human-specific pathologies and drug responses in organoids.
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It helps enable precise modeling of human-specific pathologies and drug responses in organoids.
enables targeted manipulation of sex-determining genes and regulatory elements
LiteratureIt addresses the need for targeted manipulation of pathogenic loci or regulatory elements implicated in DSD.
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It addresses the need for targeted manipulation of pathogenic loci or regulatory elements implicated in DSD.
enables targeting of root molecular pathologies underlying ischemic stroke
LiteratureThe review frames CRISPR-Cas9 as a way to more precisely target molecular drivers of ischemic stroke than current treatments. It is positioned as a neuroprotective strategy aimed at reducing neuronal damage and improving function.
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The review frames CRISPR-Cas9 as a way to more precisely target molecular drivers of ischemic stroke than current treatments. It is positioned as a neuroprotective strategy aimed at reducing neuronal damage and improving function.
enabling stable coagulation factor expression through genome integration
LiteratureIt addresses the goal of permanently correcting or compensating for F8 or F9 defects by precise editing or targeted cDNA integration.
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It addresses the goal of permanently correcting or compensating for F8 or F9 defects by precise editing or targeted cDNA integration.
enabling transgenic manipulation for optogenetic probe expression
LiteratureIt supports generation of flies expressing optogenetic probes in defined cell groups.
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It supports generation of flies expressing optogenetic probes in defined cell groups.
expands treatment options in sickle cell disease gene therapy
LiteratureIt expands treatment options for sickle cell disease within the gene-therapy landscape.
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It expands treatment options for sickle cell disease within the gene-therapy landscape.
modifying seed composition traits
LiteratureIt is used to improve protein quality traits and reduce antinutritional factors.
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It is used to improve protein quality traits and reduce antinutritional factors.
need for precise genetic engineering tools in microalgae
LiteratureIt addresses the need for targeted strain modification rather than relying only on non-specific improvement methods.
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It addresses the need for targeted strain modification rather than relying only on non-specific improvement methods.
proposed gene-editing intervention for central sensitization syndromes
LiteratureIt is proposed as a way to therapeutically edit disease-relevant biology in central sensitization syndromes. The supplied evidence does not identify a specific edited target.
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It is proposed as a way to therapeutically edit disease-relevant biology in central sensitization syndromes. The supplied evidence does not identify a specific edited target.
provides an alternative gene-editing strategy for HbF modulation
LiteratureIt is presented as an approach used to modulate HbF expression in the context of sickle cell disease.
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It is presented as an approach used to modulate HbF expression in the context of sickle cell disease.
provides a programmable gene-editing approach for PD-relevant kinase targets
LiteratureIt offers a programmable way to edit PD-associated genes, especially kinase-encoding targets discussed in the review. The review suggests this could accelerate neurodegenerative disease research and possibly therapy.
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It offers a programmable way to edit PD-associated genes, especially kinase-encoding targets discussed in the review. The review suggests this could accelerate neurodegenerative disease research and possibly therapy.
provides more precise molecular intervention than existing stroke treatments
LiteratureThe review frames CRISPR-Cas9 as a way to more precisely target molecular drivers of ischemic stroke than current treatments. It is positioned as a neuroprotective strategy aimed at reducing neuronal damage and improving function.
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The review frames CRISPR-Cas9 as a way to more precisely target molecular drivers of ischemic stroke than current treatments. It is positioned as a neuroprotective strategy aimed at reducing neuronal damage and improving function.
reduces design complexity relative to earlier nuclease platforms
LiteratureIt enables targeted genome editing and gene-function studies in poultry, including knockout-based functional genomics and trait engineering. The paper also frames it as less contentious than transgenic integration-based methods when no exogenous DNA is integrated.
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It enables targeted genome editing and gene-function studies in poultry, including knockout-based functional genomics and trait engineering. The paper also frames it as less contentious than transgenic integration-based methods when no exogenous DNA is integrated.
reducing undesirable antinutritional compounds
LiteratureIt is used to improve protein quality traits and reduce antinutritional factors.
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It is used to improve protein quality traits and reduce antinutritional factors.
supports gene delivery or engineering during CAR-T production
LiteratureIt contributes to improving production of engineered CAR-T cells.
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It contributes to improving production of engineered CAR-T cells.
supports precise genetic intervention strategies for genetically heterogeneous RP
LiteratureIt addresses the need for more precise intervention in a genetically heterogeneous retinal disease. The review positions it as part of personalized medicine for RP.
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It addresses the need for more precise intervention in a genetically heterogeneous retinal disease. The review positions it as part of personalized medicine for RP.
supports targeted modification of ion-channel content in iPSC-CMs
LiteratureIt helps build controlled arrhythmia models and supports targeted manipulation of ion-channel determinants of electrophysiology. This expands modeling beyond only naturally patient-derived variants.
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It helps build controlled arrhythmia models and supports targeted manipulation of ion-channel determinants of electrophysiology. This expands modeling beyond only naturally patient-derived variants.
Published Workflows
Objective: Test whether SWCNT- or CD-based plasmid delivery by leaf infiltration can enable reporter expression and CRISPR-Cas9 editing in cowpea, a legume described as recalcitrant for transformation.
Why it works: The abstract states that plasmid DNA delivered by SWCNTs and CDs can diffuse through plant cell walls, enabling transient expression of genetic material in plant tissues.
Stages
- 1.Reporter-gene delivery and transient expression test(functional_characterization)
This stage tests whether the nanocarrier system can deliver plasmid DNA and produce observable reporter expression in cowpea leaves.
Selection: Ability of SWCNT- or CD-based plasmid delivery to express a target gene in cowpea leaves after infiltration.
- 2.CRISPR-Cas9 delivery and editing assessment(confirmatory_validation)
This stage tests whether the nanocarrier delivery approach extends beyond transient reporter expression to CRISPR-Cas9-mediated editing.
Selection: Whether infiltrated CRISPR-Cas9 vectors targeting PDS produce editing outcomes in cowpea leaves.
Steps
- 1.Infiltrate cowpea leaves with SWCNT- or CD-delivered GUS reporter plasmidnanocarrier delivery harness
Introduce a reporter plasmid into cowpea leaf tissue to test whether the delivery system can support expression.
The abstract presents reporter-gene expression testing as the initial investigation before CRISPR-Cas9 delivery assessment.
- 2.Assess temporary GUS expression near the infiltration site by blue-color observation
Determine whether delivered reporter plasmid is transiently expressed in the surrounding infiltrated area.
This provides the stated readout for whether the nanocarrier delivery system achieved local expression before moving to CRISPR-Cas9 editing assessment.
- 3.Infiltrate cowpea leaves with CRISPR-Cas9 vectors targeting PDS using SWCNTs or CDsnanocarrier delivery harness
Test whether the nanocarrier system can deliver genome-editing constructs into cowpea leaves.
The abstract presents this as a follow-on test after reporter-gene delivery, extending the system from expression to editing.
- 4.Assess PDS editing outcomes for multiplex editing and large deletions
Determine whether delivered CRISPR-Cas9 vectors produced target-gene knockout-associated editing outcomes.
This is the confirmatory readout for whether nanocarrier-mediated delivery supports functional genome editing beyond transient expression.
Objective: Develop and apply a rapid plant assay to evaluate somatic genome editing efficiency and use it to optimize the ISAam1 TnpB nuclease.
Why it works: The workflow uses a hairy-root-transformation-based system that is described as simple, rapid, easy to implement, non-sterile, and able to visually identify transgenic hairy roots within two weeks, enabling practical evaluation of plant somatic editing activity.
Stages
- 1.Hairy-root assay system development(functional_characterization)
To create an efficient plant evaluation system for genome editing activity given variability across editing systems and target sites.
Selection: Establish a simple, rapid, and efficient plant system for evaluating somatic genome editing efficiency.
- 2.CRISPR/Cas9 validation of assay system(confirmatory_validation)
To validate the newly developed assay before applying it to ISAam1 TnpB evaluation.
Selection: Confirm the effectiveness of the hairy-root-based evaluation system using an established genome editing platform.
- 3.ISAam1 somatic editing assessment(functional_characterization)
To evaluate the plant somatic editing performance of ISAam1 after assay validation.
Selection: Assess somatic editing activity of ISAam1 TnpB in the validated plant assay system.
- 4.Protein engineering-based ISAam1 optimization(hit_picking)
To optimize ISAam1 performance in plants by finding improved variants.
Selection: Identify ISAam1 variants with enhanced somatic editing efficiency.
Steps
- 1.Develop hairy-root-transformation-based somatic editing evaluation systemassay method
Create a simple, rapid, and efficient plant assay for somatic genome editing efficiency.
An efficient evaluation system is needed because editing activity varies across genome editing systems and target sites in plants.
- 2.Validate the assay system using CRISPR/Cas9assay platform under validation
Confirm that the newly developed hairy-root assay effectively reports plant genome editing activity.
The system is validated before being applied to ISAam1 so that subsequent ISAam1 measurements are supported by an effective assay.
- 3.Assess ISAam1 somatic editing activity in the validated assayengineered nuclease evaluated in assay system
Measure somatic editing activity of ISAam1 in plants.
ISAam1 is tested after assay validation to evaluate its plant editing activity in a confirmed system.
- 4.Identify improved ISAam1 variants by protein engineeringparent nuclease and improved variants evaluated in assay
Find ISAam1 variants with higher somatic editing efficiency.
Variant optimization follows baseline ISAam1 assessment so improved versions can be identified using the established assay.
Engineering Approaches for Sustainable Protein Production in Microalgae: A Comprehensive Review.
2025Objective: Develop sustainable microalgae-based protein production platforms by combining upstream cultivation optimization, strain modification, downstream processing, extracted-protein recovery, and biorefinery integration.
Why it works: The review frames protein production as a multi-stage engineering problem in which upstream cultivation and strain modification increase biomass and protein accumulation, while downstream processing and recovery determine product quality and application range, and biorefinery integration improves economic viability.
Stages
- 1.Upstream cultivation optimization(functional_characterization)
The review states that mixotrophic cultivation is often preferred to maximize protein production and that light quality, carbon sources, and nitrogen availability direct metabolic fluxes toward protein biosynthesis.
Selection: Optimize cultivation mode and environmental inputs to maximize protein production and biomass yield.
- 2.Strain modification(functional_characterization)
The abstract identifies CRISPR/Cas9 as promising but still challenging and limited for enhancing microalgal protein production, while random mutagenesis is described as proven effective across multiple strains.
Selection: Apply genetic or mutational approaches to improve strains for protein accumulation.
- 3.Whole-cell downstream processing(secondary_characterization)
The abstract emphasizes drying, extrusion forming, and fermentation as downstream engineering approaches for improving whole-cell product properties.
Selection: Use downstream processing methods to improve nutritional and functional properties of whole-cell microalgal protein products for food and feed.
- 4.Extracted-protein recovery and quality shaping(secondary_characterization)
The abstract states that extracted proteins broaden potential applications and that their quality is significantly affected by cell disruption/extraction, purification, and hydrolysis methods.
Selection: Choose extraction, purification, and hydrolysis methods that support desired quality in extracted microalgal proteins.
- 5.Protein-first biorefinery integration(decision_gate)
The abstract states that novel biorefinery strategies enhance economic viability by integrating value-added biomass utilization within a protein-first recovery scheme.
Selection: Integrate value-added biomass utilization around a protein-first recovery scheme to improve economic viability.
Objective: Faithfully recapitulate in vitro arrhythmogenic events in a high-throughput manner for personalized arrhythmia monitoring and drug screening.
Why it works: The review proposes that combining patient-relevant iPSC-CM models with targeted engineering, more physiologic 3D culture formats, and non-invasive high-throughput optical phenotyping can jointly improve fidelity and scalability of arrhythmia modeling.
Stages
- 1.Establish patient-specific or engineered iPSC-CM arrhythmia models(library_design)
The review states that traditional animal models and heterologous cell cultures do not truly reflect human cardiac electro-pathophysiology, motivating patient-specific and variant-defined iPSC-CM models.
Selection: Use patient-specific genetic background or engineered variants to create arrhythmia-relevant iPSC-CM models.
- 2.Gene-editing-based model engineering and ion-channel modification(library_build)
The review describes CRISPR/Cas9 as enabling patient-independent and variant-induced iPSC-CM arrhythmia models and later links gene editing to maturity improvement through ion-channel modification.
Selection: Apply CRISPR/Cas9 or related editing to establish patient-independent or variant-induced models and modify specific ion channels.
- 3.Upgrade model format with 3D iPSC-CM culture(functional_characterization)
The review explicitly states that novel 3D iPSC-CM models can better capture in vitro characteristics.
Selection: Use novel 3D iPSC-CM models to better capture in vitro characteristics.
- 4.All-optical electrophysiological phenotyping for variant stratification and drug discovery(confirmatory_validation)
The review states that all-optical platforms provide non-invasive and high-throughput electrophysiological data useful for stratification of emerging arrhythmogenic variants and drug discovery.
Selection: Collect non-invasive, high-throughput electrophysiological data using all-optical platforms.
Objective: Generate and analyze Drosophila models carrying disease-associated epilepsy variants in order to interpret pathogenic consequences, study seizure-related phenotypes, and explore therapeutic implications.
Why it works: The review abstract describes a linked workflow in which disease-associated variants are introduced into flies, then screened for organism-level seizure-related phenotypes and drug responses, and further interrogated with optogenetic and imaging methods to trace functional alterations.
Stages
- 1.Generate flies carrying disease-associated variants(library_build)
This stage creates the fly models needed for downstream epilepsy phenotyping and functional analysis.
Selection: Introduction of disease-associated variants into Drosophila using gene editing techniques such as CRISPR/Cas9.
- 2.Screen variant-carrying flies for seizure-related phenotypes and drug responses(broad_screen)
This stage identifies observable consequences of disease-associated variants and potential therapeutic-response patterns.
Selection: Phenotypic and behavioral abnormalities, shifting of seizure thresholds, and response to anti-seizure medications and other substances.
- 3.Manipulate neuronal activity and induce seizures with optogenetic tools(functional_characterization)
This stage provides controlled perturbation of neural activity to probe seizure mechanisms beyond passive phenotype observation.
Selection: Ability to modify neuronal activity and induce seizures using optogenetic tools.
- 4.Trace mutation-associated functional alterations with calcium and fluorescent imaging(secondary_characterization)
This stage adds mechanistic readouts that can connect epilepsy-gene mutations to functional changes.
Selection: Imaging-based tracing of functional alterations caused by mutations in epilepsy genes.
Objective: Generate transgenic Drosophila that express optogenetic probes in specific cell groups and use them in basic neuroscience experiments.
Why it works: The review frames Drosophila optogenetics as depending on first generating transgenic flies and then using binary expression systems to restrict probe expression to specific cells before running optogenetic experiments.
Stages
- 1.Generate transgenic flies(library_build)
The review states that transgenic techniques are crucial for Drosophila optogenetics and describes generating flies that express optogenetic probes in specific cells.
Selection: Establish flies expressing optogenetic probes
- 2.Specify cell-targeted expression(functional_characterization)
Binary expression systems are described specifically in the context of cell-specific gene expression techniques.
Selection: Use binary expression systems for cell-specific gene expression
- 3.Run basic larval optogenetic experiment(confirmatory_validation)
The review states that it presents a short and basic optogenetic experiment with Drosophila larvae as a practical example.
Selection: Practical example of optogenetic use in Drosophila larvae
Taxonomy & Function
Primary hierarchy
Mechanism Branch
Architecture: A composed arrangement of multiple parts that instantiates one or more mechanisms.
Mechanisms
double-stranded dna break inductionPhotocleavageprogrammable dna cleavagesite-specific genome modificationTranslation ControlTarget processes
editingrecombinationselectiontranslationInput: Chemical
Implementation Constraints
The evidence identifies CRISPR/Cas9 as a bacterial type II CRISPR/Cas9 system engineered for use in mammalian cells. No specific construct architecture, guide design rules, delivery modality, cofactors, or expression system details are provided in the supplied text.
The supplied evidence does not provide specific quantitative performance metrics, off-target profiles, delivery constraints, or comparative limitations. Although cancer therapy and gene therapy applications are mentioned, the excerpts do not document detailed validation outcomes or boundary conditions.
Validation
Supporting Sources
Ranked Claims
KSHV-associated malignancies lack virus-specific targeted treatments and current clinical outcomes remain suboptimal, especially in immunocompromised patients.
The review compares prime editing with CRISPR-Cas9 and Base editing as gene-editing strategies for HbF modulation.
This review also provides a comparative overview of prime editing and other gene-editing strategies for HbF modulation, such as CRISPR-Cas9 and Base editing.
Viral vectors, nanocarriers, and extracellular vesicles are described as important CRISPR delivery systems for achieving access to neural cells.
Various CRISPR delivery systems, including viral vectors, nanocarriers, and extracellular vesicles, play crucial roles in the effective access of this tool to neural cells.
LANA immunostaining and histopathology have important limitations in early or atypical lesions and in distinguishing latent from lytic infection.
Histopathology and LANA immunostaining remain the diagnostic standard and gold standard for current KS diagnosis.
Prime editing has emerged as an experimental approach capable of introducing multiple HPFH-like mutations within b3-globin promoters.
Regarding advances in b3-globin editing, "prime editing", although still in the experimental phase, has recently emerged as an innovative approach capable of introducing multiple HPFH-like mutations within b3-globin promoters...
Promoter engineering and Prime Editing are expected to improve precision of soybean genetic modification and minimize pleiotropic effects.
The integration of new approaches, such as promoter engineering and Prime Editing, promises to further enhance the precision of genetic modifications, minimizing pleiotropic effects.
Editing SWEET10a and SWEET10b allows modulation of the soybean oil-protein balance.
the editing of sugar transporters SWEET10a and SWEET10b allows the modulation of the oil-protein balance
Inactivation of genes related to antinutritional factors has reduced expression of phytate and protease inhibitors in soybean.
Simultaneously, the inactivation of genes related to antinutritional factors has significantly reduced the expression of compounds such as phytate and protease inhibitors.
Silencing negative regulatory genes such as CIF1 and AIP2 can elevate soybean seed protein content.
Recent studies demonstrate that the silencing of negative regulatory genes, such as CIF1 and AIP2, can elevate the protein content of seeds
Targeted and safe delivery remains a persistent challenge for CRISPR-based therapy in ischemic stroke.
Despite persistent challenges in targeted and safe delivery, substantial preclinical advances, primarily in rodent models, underscore the potential for CRISPR-based therapies to transform future stroke treatment.
Reviewed studies report that CRISPR-Cas9 modulation of inflammation, oxidative stress, and cell-death pathways can prevent neuronal damage and improve neurological function in ischemic stroke contexts.
Studies have shown that the use of CRISPR-Cas9 to modulate key pathogenic pathways, including those governing inflammation, oxidative stress, and cell death, can prevent neuronal damage and improve neurological function.
Programmable nucleases including CRISPR/Cas9, TALENs, and ZFNs induce double-stranded DNA breaks at specific sites, enabling precise correction or targeted transgene integration.
This approach involves the use of programmable nucleases (CRISPR/Cas9, TALENs, ZFNs) that induce double-stranded DNA breaks at specific sites, allowing precise correction or targeted transgene integration.
CRISPR-Cas9 is presented as a next-generation approach that aims to inhibit viral replication, modulate oncogenic pathways, and enhance immune responses in KSHV-associated disease.
Gene editing for hemophilia is presented as an emerging approach that aims to provide a permanent cure by precise correction of the mutated gene or targeted integration of coagulation factor cDNA for stable expression.
Gene editing for hemophilia is an emerging approach that aims to provide a permanent cure by editing the mutated gene precisely or targeted integration of coagulation factor cDNA into the host genome for stable expression.
RNAi, CRISPR/Cas9, and AlphaFold2-guided gene editing are used to modify genes involved in carbon and nitrogen metabolism and storage proteins in soybean.
This work reviews the main progress achieved through transgenesis, induced mutagenesis, and precision gene editing, highlighting the role of tools such as RNAi, CRISPR/Cas9, and AlphaFold2-guided gene editing in modifying genes involved in carbon and nitrogen metabolism and storage proteins.
AAV-based vectors including AAV8 and AAVDJ effectively delivered genes such as Lhcgr into testicular interstitial tissues and restored testosterone synthesis and fertility in mouse models.
AAV-based vectors-including AAV8 and synthetic AAVDJ-have demonstrated effective delivery of genes like Lhcgr into testicular interstitial tissues, restoring testosterone synthesis and fertility in mouse models.
Lentiviral vectors enabled stable overexpression of SOX9 or NR5A1 in hiPSCs, inducing differentiation into Sertoli-like and Leydig-like cells with partial restoration of testicular function in vitro.
Lentiviral vectors have enabled stable overexpression of transcriptional regulators (e.g., SOX9, NR5A1) in hiPSCs, inducing differentiation into Sertoli- and Leydig-like cells, with partial restoration of testicular function in vitro.
The PaGZ-1-Aiia variant inhibited biofilm formation and disrupted established biofilms.
the PaGZ-1-Aiia variant showed significant promise in both inhibiting biofilm formation and disrupting established biofilms
CRISPR/Cas9-mediated gene editing enables precise genetic modification in soybean and has produced improved oil composition, increased isoflavone content, and resistance to biotic stresses.
Lentiviral vector-mediated gene addition and CRISPR/Cas9 gene editing offer curative potential for sickle cell disease.
Recent advances in gene therapy have transformed the therapeutic landscape of SCD, offering curative potential through techniques such as lentiviral vector-mediated gene addition and CRISPR/Cas9 gene editing.
Marker-assisted selection using SSRs and SNPs facilitates efficient identification and incorporation of desired soybean traits including disease resistance, abiotic stress tolerance, and improved seed quality.
RNA interference modulates gene expression in soybean to optimize nutritional properties and stress responses.
CAR-T cell therapy has shown success in hematological malignancies but faces significant challenges in solid tumors such as colorectal cancer.
Current challenges in developing heat-tolerant rice include integrating regulatory mechanisms, developing realistic heat simulation systems, validating candidate-gene functionality, and managing trait trade-offs.
Finally, we address current challenges, including integrating regulatory mechanisms, developing realistic heat simulation systems, validating the functionality of candidate genes, and managing trait trade-offs.
The review identifies long-term genetic stability, scalability, and off-target effects as challenges for genetically engineered tissues.
We address the field's challenges, including long-term genetic stability, scalability, and off-target effects, while also considering the ethical implications and evolving regulatory landscape of genetically engineered tissues.
Clinical trial outcomes for emerging sickle cell disease gene therapies are encouraging, including reduced vaso-occlusive crises and transfusion independence.
While clinical trial outcomes are encouraging, with reduced vaso-occlusive crises and transfusion independence, major challenges remain
These molecular breeding approaches overcome limitations of traditional methods by shortening the breeding cycle and allowing simultaneous improvement of multiple traits.
The engineered PaGZ-1 phages showed superior inhibition of biofilm formation compared with the wild-type phage.
The resulting engineered phages demonstrated superior inhibition of biofilm formation compared to the wild-type phage.
Electroporation and other non-viral delivery methods may offer safer gene editing for HSCs but require further optimization.
Lentiviral vectors were the most common delivery method in the reviewed studies, but insertional mutagenesis remains a concern.
Plasmid-based ARG expression hinders stable in vivo expression and limits space for inserting drug-release regulatory components.
ARGs are often tested on plasmids, which hinders stable expression in vivo and limits the space for inserting components that regulate drug release.
Drying, extrusion forming, and fermentation are emphasized as downstream engineering approaches for improving the nutritional and functional properties of whole-cell microalgal proteins for food and feed applications.
Two T0 lines, HL40 and HL64, showed successful edits in all seven target genes.
Two T0 lines (HL40 and HL64) exhibited successful edits in all seven target genes, with mutations consisting of single-base insertions and deletions up to 26 bp.
Base editing avoids the need for HDR but still faces delivery challenges in HSC applications.
CRISPR/Cas9 provides precise editing in HSCs but is limited by low HDR efficiency in quiescent HSCs.
Random mutagenesis has been proven effective in improving multiple microalgal strains for increased protein accumulation.
The review describes base editing and synthetic genetic circuits as emerging technologies explored for creating smart tissues capable of dynamic environmental responses.
Emerging technologies in genetic engineering, including base editing and synthetic genetic circuits, have been explored for their potential to create "smart" tissues capable of dynamic environmental responses.
The explored improvement strategies aim to enhance CAR-T cell specificity, improve resistance to immunosuppressive signals, and optimize in vivo functionality.
CRISPR-Cas9 was used to engineer the P. aeruginosa phage PaGZ-1 to express Aiia or a phage-derived depolymerase.
we then used CRISPR-Cas9 to engineer the P. aeruginosa phage PaGZ-1 to express these biofilm-disrupting genes
CRISPR-Cas9-mediated genome insertion of ARGs with promoter and copy-number optimization produced ultrasound-visible engineered bacteria expressing gas vesicles from the genome.
By using CRISPR-Cas9 technology, we inserted ARGs into the genome and optimized the promoter strength and copy number for ARG expression, constructing ultrasound-visible engineered bacteria expressing gas vesicles on the genome.
Dual CARs, tandem CARs, SynNotch systems, and universal or inhibitory CARs expand antigen targeting and reduce off-tumor toxicity.
novel engineering strategies such as dual CARs, tandem CARs, SynNotch systems, and universal or inhibitory CARs have expanded antigen targeting and reduced off-tumor toxicity
Emerging gene editing approaches such as CRISPR/Cas9 are expanding treatment options and moving sickle cell disease gene therapy into clinical application.
Emerging gene editing approaches such as CRISPR/Cas9 are expanding treatment options, marking the transition of SCD gene therapy from theoretical concept to clinical application.
CAR-engineered HSCs showed durable tumor clearance and multilineage immune reconstitution in the reviewed preclinical evidence.
CRISPR/Cas9 has been used in vitro for gene correction or epigenetic activation, including SRY promoter demethylation in embryonic stem cells, and for targeted disruption of SOX9 enhancers in mice to model 46,XX testicular DSD.
CRISPR/Cas9 has been utilized to correct or epigenetically activate gene expression in vitro, such as SRY promoter demethylation in embryonic stem cells, and targeted disruption of SOX9 enhancers to model 46, XX testicular DSD in mice.
The constructed ultrasound-visible engineered bacteria can stably synthesize gas vesicles, output ultrasound signals, and directly carry drug plasmids for tumor therapy.
The constructed ultrasound-visible engineered bacteria can stably synthesize gas vesicles and output ultrasound signals while directly carrying drug plasmids for tumor therapy.
Heterozygous alleles segregated to homozygous genotypes in T2 and this was accompanied by confirmed loss of enzymatic activity.
Heterozygous alleles segregated into homozygous genotypes in the T2 generation, accompanied by confirmed loss of enzymatic activity.
Emerging technologies including organoid fusion, xenografting, and optogenetics are expected to enhance understanding of cellular interactions and microenvironmental dynamics.
Major challenges for emerging sickle cell disease gene therapies include high costs, the need for myeloablative conditioning, and limited access in high-burden regions.
major challenges remain, including high costs, need for myeloablative conditioning, and limited access in high-burden regions
The review states that integrating genetic engineering with 3D-bioprinting, microfluidics, and smart biomaterials expands the horizons of complex tissue fabrication.
We further investigate the integration of these genetic approaches with emerging technologies such as 3D-bioprinting, microfluidics, and smart biomaterials, which collectively expand the horizons of complex tissue fabrication.
Major challenges for CAR-T therapy in colorectal cancer include antigen heterogeneity, an immunosuppressive tumor microenvironment, and on-target off-tumor toxicity.
The emerging gene-therapy approaches discussed aim to restore normal hemoglobin production or reactivate fetal hemoglobin expression.
These approaches aim to restore normal hemoglobin production or reactivate fetal hemoglobin expression.
Multiplex CRISPR/Cas9 was applied in Nicotiana benthamiana to simultaneously target five α-1,3-fucosyltransferase genes and two β-1,2-xylosyltransferase genes.
We applied multiplex CRISPR/Cas9 genome editing in Nicotiana benthamiana to simultaneously target five α-1,3-fucosyltransferase genes and two β-1,2-xylosyltransferase genes.
The study provides a straightforward method for introducing exogenous genes into non-model P. aeruginosa phage genomes.
Our findings provide a straightforward method for introducing exogenous genes into non-model P. aeruginosa phage genomes
Chimeric antigen receptor T cells, tumor-infiltrating lymphocytes, and T cell receptor-engineered T cells are key adoptive cell therapy modalities in colorectal cancer discussion.
The work establishes the first Nicotiana benthamiana lines reported to be fully Cas9-free and homozygously edited at all seven key glycosyltransferase loci.
This work establishes the first N. benthamiana lines that are fully Cas9-free and homozygously edited at all seven key glycosyltransferase loci.
Genomic selection improves prediction accuracy for complex quantitative soybean traits such as yield by integrating genome-wide molecular markers with phenotypic data.
Knocking out htrA in VNP20009 increased the maximum injection dose tenfold and tumor specificity hundredfold.
by knocking out the stress protein gene htrA in VNP20009, we increased the maximum injection dose by tenfold and the tumor specificity by a hundredfold.
T2 plants showed no detectable morphological or growth differences compared with wild-type plants.
T2 plants showed no detectable morphological or growth differences compared with wild-type plants, indicating no adverse phenotypic effects.
Mixotrophic cultivation is often preferred for maximizing microalgal protein production because it enables significantly higher biomass yields.
Viral vectors, transposons, CRISPR/Cas9, and RNA-based electroporation are emerging gene delivery technologies that improve CAR-T production.
Emerging gene delivery technologies, including viral vectors, transposons, CRISPR/Cas9, and RNA-based electroporation, are improving CAR-T production.
The quality of extracted microalgal proteins is significantly affected by the methods used for cell disruption/extraction, purification, and hydrolysis.
The review examines CRISPR-Cas9, TALENs, and synthetic biology as genetic engineering approaches for modifying cellular behaviors and functions in tissue engineering.
We critically examine the application of advanced genetic engineering techniques, including CRISPR-Cas9, TALENs, and synthetic biology, in modifying cellular behaviors and functions for tissue engineering.
Integration of genetic tools such as CRISPR-Cas9, prime editing, and lineage tracing has facilitated precise modeling of human-specific pathologies and drug responses in organoids.
Suicide gene strategies were effective in mitigating safety risks in the reviewed HSC engineering context.
The study identified stable Cas9-free homozygous Nicotiana benthamiana lines with mutations across all targeted loci and ultimately generated 12 independent lines.
In later generations, we identified stable Cas9-free homozygous lines containing mutations across all targeted loci... Ultimately, we generated 12 independent Cas9-free, glycoengineered, homozygous lines.
Combinatorial approaches including immune checkpoint inhibitors, cytokines, and CRISPR/Cas9 are being explored to address CAR-T limitations in colorectal cancer.
Multi-omics integration, CRISPR/Cas9 genome editing, marker-assisted selection, and rational design breeding have recent applications in enhancing heat-tolerant rice varieties.
Additionally, we summarize recent applications of cutting-edge technologies in the enhancement of heat-tolerant rice varieties, including multi-omics integration, CRISPR/Cas9 genome editing, marker-assisted selection (MAS), and rational design breeding.
The paper frames CRISPR/Cas9 delivered by nanoparticle-based non-viral approaches as a potential nanotherapy direction for rare central sensitization syndromes.
Title: CRISPR-guided nanotherapy for rare central sensitization syndromes. Web research summary: the anchor PMC full text explicitly frames the topic around CRISPR/Cas9 as a potential CNS therapeutic modality and nanoparticle-based non-viral delivery for CRISPR.
Clinical trials using lentiviral vector-mediated gene insertion showed promising therapeutic outcomes by preventing hemoglobin aggregation in sickle cell disease.
Clinical trials utilizing lentiviral vector-mediated gene insertion have demonstrated promising therapeutic outcomes by preventing hemoglobin aggregation.
CRISPR/Cas9 is a promising precise gene editing tool in microalgae, but its application to enhancing microalgal protein production remains challenging and limited.
CRISPR/Cas9 is described as a convenient way to generate flies carrying disease-associated variants.
Gene editing techniques, such as CRISPR/Cas9, are a convenient way to generate flies carrying disease-associated variants.
Calcium and fluorescent imaging can be used to trace functional alterations caused by mutations in epilepsy genes.
In combination with calcium and fluorescent imaging, functional alterations caused by mutations in epilepsy genes can be traced.
Bang-sensitive mutants respond to mechanical stimulation such as a brief vortex with stereotypic seizures and paralysis.
These flies respond to mechanical stimulation, such as a brief vortex, with stereotypic seizures and paralysis.
Variant-carrying flies can be screened for phenotypic and behavioral abnormalities, seizure-threshold shifts, and responses to anti-seizure medications and other substances.
These flies can be screened for phenotypic and behavioral abnormalities, shifting of seizure thresholds, and response to anti-seizure medications and other substances.
Effective delivery vectors, CRISPR/Cas9 technology, and iPSC-based cell transplantation are described as accelerating personalized precision medicine in RP.
Specifically, technologies, such as effective delivery vectors, CRISPR/Cas9 technology, and iPSC-based cell transplantation, hasten the pace of personalized precision medicine in RP.
By 2013, CRISPR/Cas9 systems had been engineered to allow gene editing in mammalian cells.
By 2013, CRISPR/Cas9 systems had been engineered to allow gene editing in mammalian cells.
By 2013, CRISPR/Cas9 systems had been engineered to allow gene editing in mammalian cells.
By 2013, CRISPR/Cas9 systems had been engineered to allow gene editing in mammalian cells.
By 2013, CRISPR/Cas9 systems had been engineered to allow gene editing in mammalian cells.
By 2013, CRISPR/Cas9 systems had been engineered to allow gene editing in mammalian cells.
By 2013, CRISPR/Cas9 systems had been engineered to allow gene editing in mammalian cells.
By 2013, CRISPR/Cas9 systems had been engineered to allow gene editing in mammalian cells.
By 2013, CRISPR/Cas9 systems had been engineered to allow gene editing in mammalian cells.
By 2013, CRISPR/Cas9 systems had been engineered to allow gene editing in mammalian cells.
By 2013, CRISPR/Cas9 systems had been engineered to allow gene editing in mammalian cells.
By 2013, CRISPR/Cas9 systems had been engineered to allow gene editing in mammalian cells.
By 2013, CRISPR/Cas9 systems had been engineered to allow gene editing in mammalian cells.
By 2013, CRISPR/Cas9 systems had been engineered to allow gene editing in mammalian cells.
Programmable nucleases such as CRISPR/Cas9 expanded gene therapy applications from semi-random gene addition to site-specific genome modification.
With the advent of novel programmable nucleases, such as CRISPR/Cas9, it has been possible to expand the applications of gene therapy beyond semi-random gene addition to site-specific modification of the genome
CRISPR/Cas9 and related systems are described as designer nucleases of choice because of ease of design, low cytotoxicity, and increased efficiency.
The ease of design, low cytotoxicity, and increased efficiency have made CRISPR/Cas9 and its related systems the designer nucleases of choice for many.
CRISPR/Cas9 and related systems are described as designer nucleases of choice because of ease of design, low cytotoxicity, and increased efficiency.
The ease of design, low cytotoxicity, and increased efficiency have made CRISPR/Cas9 and its related systems the designer nucleases of choice for many.
CRISPR/Cas9 and related systems are described as designer nucleases of choice because of ease of design, low cytotoxicity, and increased efficiency.
The ease of design, low cytotoxicity, and increased efficiency have made CRISPR/Cas9 and its related systems the designer nucleases of choice for many.
CRISPR/Cas9 and related systems are described as designer nucleases of choice because of ease of design, low cytotoxicity, and increased efficiency.
The ease of design, low cytotoxicity, and increased efficiency have made CRISPR/Cas9 and its related systems the designer nucleases of choice for many.
CRISPR/Cas9 and related systems are described as designer nucleases of choice because of ease of design, low cytotoxicity, and increased efficiency.
The ease of design, low cytotoxicity, and increased efficiency have made CRISPR/Cas9 and its related systems the designer nucleases of choice for many.
CRISPR/Cas9 and related systems are described as designer nucleases of choice because of ease of design, low cytotoxicity, and increased efficiency.
The ease of design, low cytotoxicity, and increased efficiency have made CRISPR/Cas9 and its related systems the designer nucleases of choice for many.
CRISPR/Cas9 and related systems are described as designer nucleases of choice because of ease of design, low cytotoxicity, and increased efficiency.
The ease of design, low cytotoxicity, and increased efficiency have made CRISPR/Cas9 and its related systems the designer nucleases of choice for many.
ZFN and TALEN provided sequence-specific gene-editing capacity but their broad utility was limited by laborious nuclease design and synthesis, limited target choices, and poor editing efficiency.
While these approaches provided sequence-specific gene-editing capacity, the laborious process of designing and synthesizing recombinant nucleases to recognize a specific target sequence, combined with limited target choices and poor editing efficiency, ultimately minimized the broad utility of these systems.
ZFN and TALEN provided sequence-specific gene-editing capacity but their broad utility was limited by laborious nuclease design and synthesis, limited target choices, and poor editing efficiency.
While these approaches provided sequence-specific gene-editing capacity, the laborious process of designing and synthesizing recombinant nucleases to recognize a specific target sequence, combined with limited target choices and poor editing efficiency, ultimately minimized the broad utility of these systems.
ZFN and TALEN provided sequence-specific gene-editing capacity but their broad utility was limited by laborious nuclease design and synthesis, limited target choices, and poor editing efficiency.
While these approaches provided sequence-specific gene-editing capacity, the laborious process of designing and synthesizing recombinant nucleases to recognize a specific target sequence, combined with limited target choices and poor editing efficiency, ultimately minimized the broad utility of these systems.
ZFN and TALEN provided sequence-specific gene-editing capacity but their broad utility was limited by laborious nuclease design and synthesis, limited target choices, and poor editing efficiency.
While these approaches provided sequence-specific gene-editing capacity, the laborious process of designing and synthesizing recombinant nucleases to recognize a specific target sequence, combined with limited target choices and poor editing efficiency, ultimately minimized the broad utility of these systems.
ZFN and TALEN provided sequence-specific gene-editing capacity but their broad utility was limited by laborious nuclease design and synthesis, limited target choices, and poor editing efficiency.
While these approaches provided sequence-specific gene-editing capacity, the laborious process of designing and synthesizing recombinant nucleases to recognize a specific target sequence, combined with limited target choices and poor editing efficiency, ultimately minimized the broad utility of these systems.
ZFN and TALEN provided sequence-specific gene-editing capacity but their broad utility was limited by laborious nuclease design and synthesis, limited target choices, and poor editing efficiency.
While these approaches provided sequence-specific gene-editing capacity, the laborious process of designing and synthesizing recombinant nucleases to recognize a specific target sequence, combined with limited target choices and poor editing efficiency, ultimately minimized the broad utility of these systems.
ZFN and TALEN provided sequence-specific gene-editing capacity but their broad utility was limited by laborious nuclease design and synthesis, limited target choices, and poor editing efficiency.
While these approaches provided sequence-specific gene-editing capacity, the laborious process of designing and synthesizing recombinant nucleases to recognize a specific target sequence, combined with limited target choices and poor editing efficiency, ultimately minimized the broad utility of these systems.
Transgenic techniques are crucial for applying optogenetics in Drosophila neuroscience.
Gal4-UAS, LexA-lexAop, and Q-system are binary expression systems used for cell-specific gene expression in Drosophila optogenetics.
CRISPR and Cas proteins were identified as part of a microbial adaptive immune system that targets phage DNA to fight bacteriophage reinfection.
CRISPR and the CRISPR-associated (Cas) proteins were identified as part of the microbial adaptive immune system, by targeting phage DNA, to fight bacteriophage reinfection.
CRISPR and Cas proteins were identified as part of a microbial adaptive immune system that targets phage DNA to fight bacteriophage reinfection.
CRISPR and the CRISPR-associated (Cas) proteins were identified as part of the microbial adaptive immune system, by targeting phage DNA, to fight bacteriophage reinfection.
CRISPR and Cas proteins were identified as part of a microbial adaptive immune system that targets phage DNA to fight bacteriophage reinfection.
CRISPR and the CRISPR-associated (Cas) proteins were identified as part of the microbial adaptive immune system, by targeting phage DNA, to fight bacteriophage reinfection.
CRISPR and Cas proteins were identified as part of a microbial adaptive immune system that targets phage DNA to fight bacteriophage reinfection.
CRISPR and the CRISPR-associated (Cas) proteins were identified as part of the microbial adaptive immune system, by targeting phage DNA, to fight bacteriophage reinfection.
CRISPR and Cas proteins were identified as part of a microbial adaptive immune system that targets phage DNA to fight bacteriophage reinfection.
CRISPR and the CRISPR-associated (Cas) proteins were identified as part of the microbial adaptive immune system, by targeting phage DNA, to fight bacteriophage reinfection.
CRISPR and Cas proteins were identified as part of a microbial adaptive immune system that targets phage DNA to fight bacteriophage reinfection.
CRISPR and the CRISPR-associated (Cas) proteins were identified as part of the microbial adaptive immune system, by targeting phage DNA, to fight bacteriophage reinfection.
CRISPR and Cas proteins were identified as part of a microbial adaptive immune system that targets phage DNA to fight bacteriophage reinfection.
CRISPR and the CRISPR-associated (Cas) proteins were identified as part of the microbial adaptive immune system, by targeting phage DNA, to fight bacteriophage reinfection.
The review discusses various CRISPR systems and their broad utility in genome manipulation, including how CRISPR-controlled modification of DNA repair genes has advanced understanding of genome stability mechanisms.
In this review, we discuss the various CRISPR systems and their broad utility in genome manipulation. We will explore how CRISPR-controlled modifications have advanced our understanding of the mechanisms of genome stability, using the modulation of DNA repair genes as examples.
The review discusses various CRISPR systems and their broad utility in genome manipulation, including how CRISPR-controlled modification of DNA repair genes has advanced understanding of genome stability mechanisms.
In this review, we discuss the various CRISPR systems and their broad utility in genome manipulation. We will explore how CRISPR-controlled modifications have advanced our understanding of the mechanisms of genome stability, using the modulation of DNA repair genes as examples.
The review discusses various CRISPR systems and their broad utility in genome manipulation, including how CRISPR-controlled modification of DNA repair genes has advanced understanding of genome stability mechanisms.
In this review, we discuss the various CRISPR systems and their broad utility in genome manipulation. We will explore how CRISPR-controlled modifications have advanced our understanding of the mechanisms of genome stability, using the modulation of DNA repair genes as examples.
The review discusses various CRISPR systems and their broad utility in genome manipulation, including how CRISPR-controlled modification of DNA repair genes has advanced understanding of genome stability mechanisms.
In this review, we discuss the various CRISPR systems and their broad utility in genome manipulation. We will explore how CRISPR-controlled modifications have advanced our understanding of the mechanisms of genome stability, using the modulation of DNA repair genes as examples.
The review discusses various CRISPR systems and their broad utility in genome manipulation, including how CRISPR-controlled modification of DNA repair genes has advanced understanding of genome stability mechanisms.
In this review, we discuss the various CRISPR systems and their broad utility in genome manipulation. We will explore how CRISPR-controlled modifications have advanced our understanding of the mechanisms of genome stability, using the modulation of DNA repair genes as examples.
The review discusses various CRISPR systems and their broad utility in genome manipulation, including how CRISPR-controlled modification of DNA repair genes has advanced understanding of genome stability mechanisms.
In this review, we discuss the various CRISPR systems and their broad utility in genome manipulation. We will explore how CRISPR-controlled modifications have advanced our understanding of the mechanisms of genome stability, using the modulation of DNA repair genes as examples.
The review discusses various CRISPR systems and their broad utility in genome manipulation, including how CRISPR-controlled modification of DNA repair genes has advanced understanding of genome stability mechanisms.
In this review, we discuss the various CRISPR systems and their broad utility in genome manipulation. We will explore how CRISPR-controlled modifications have advanced our understanding of the mechanisms of genome stability, using the modulation of DNA repair genes as examples.
Site-specific genome modification is presented as holding promise for safer genetic manipulation.
site-specific modification of the genome, holding the promise for safer genetic manipulation
Site-specific genome modification is presented as holding promise for safer genetic manipulation.
site-specific modification of the genome, holding the promise for safer genetic manipulation
Site-specific genome modification is presented as holding promise for safer genetic manipulation.
site-specific modification of the genome, holding the promise for safer genetic manipulation
Site-specific genome modification is presented as holding promise for safer genetic manipulation.
site-specific modification of the genome, holding the promise for safer genetic manipulation
Site-specific genome modification is presented as holding promise for safer genetic manipulation.
site-specific modification of the genome, holding the promise for safer genetic manipulation
Site-specific genome modification is presented as holding promise for safer genetic manipulation.
site-specific modification of the genome, holding the promise for safer genetic manipulation
Site-specific genome modification is presented as holding promise for safer genetic manipulation.
site-specific modification of the genome, holding the promise for safer genetic manipulation
Clinical translation of gene editing in human HSPCs faces current challenges despite potential advantages.
We highlight the potential advantages and the current challenges toward safe and effective clinical translation of gene editing for the treatment of hematological diseases.
Clinical translation of gene editing in human HSPCs faces current challenges despite potential advantages.
We highlight the potential advantages and the current challenges toward safe and effective clinical translation of gene editing for the treatment of hematological diseases.
Clinical translation of gene editing in human HSPCs faces current challenges despite potential advantages.
We highlight the potential advantages and the current challenges toward safe and effective clinical translation of gene editing for the treatment of hematological diseases.
Clinical translation of gene editing in human HSPCs faces current challenges despite potential advantages.
We highlight the potential advantages and the current challenges toward safe and effective clinical translation of gene editing for the treatment of hematological diseases.
Clinical translation of gene editing in human HSPCs faces current challenges despite potential advantages.
We highlight the potential advantages and the current challenges toward safe and effective clinical translation of gene editing for the treatment of hematological diseases.
Clinical translation of gene editing in human HSPCs faces current challenges despite potential advantages.
We highlight the potential advantages and the current challenges toward safe and effective clinical translation of gene editing for the treatment of hematological diseases.
Clinical translation of gene editing in human HSPCs faces current challenges despite potential advantages.
We highlight the potential advantages and the current challenges toward safe and effective clinical translation of gene editing for the treatment of hematological diseases.
Gene therapy can result in stable or inducible transgene expression and can allow nearly specific expression in target cells.
HSV-1-derived viral vectors are presented as potential tools for simultaneous delivery and expression of multiple transgene cassettes.
Cell-specific and inducible promoters allow gene products to be expressed only in specific cells and allow control of transcriptional activation.
DREADDs are presented as systems allowing spatial or temporal control of expression in CNS disease applications.
RNA interference is presented as a post-transcriptional regulation system applied to CNS diseases.
CRISPR/Cas9 and zinc finger proteins are included as gene-editing technologies relevant to CNS disease applications.
CRISPR/Cas9 rapidly became an essential component of research on apicomplexan parasites after its first reported application in this group.
this technology has rapidly become an essential component of research on apicomplexan parasites
CRISPR-Cas9 can be used to rapidly engineer immune cells and oncolytic viruses for cancer immunotherapeutic applications.
CRISPR-Cas9 can be employed to rapidly engineer immune cells and oncolytic viruses for cancer immunotherapeutic applications.
CRISPR-Cas9 can be used to rapidly engineer immune cells and oncolytic viruses for cancer immunotherapeutic applications.
CRISPR-Cas9 can be employed to rapidly engineer immune cells and oncolytic viruses for cancer immunotherapeutic applications.
CRISPR-Cas9 can be used to rapidly engineer immune cells and oncolytic viruses for cancer immunotherapeutic applications.
CRISPR-Cas9 can be employed to rapidly engineer immune cells and oncolytic viruses for cancer immunotherapeutic applications.
CRISPR-Cas9 can be used to rapidly engineer immune cells and oncolytic viruses for cancer immunotherapeutic applications.
CRISPR-Cas9 can be employed to rapidly engineer immune cells and oncolytic viruses for cancer immunotherapeutic applications.
CRISPR-Cas9 can be used to rapidly engineer immune cells and oncolytic viruses for cancer immunotherapeutic applications.
CRISPR-Cas9 can be employed to rapidly engineer immune cells and oncolytic viruses for cancer immunotherapeutic applications.
CRISPR-Cas9 can be used to rapidly engineer immune cells and oncolytic viruses for cancer immunotherapeutic applications.
CRISPR-Cas9 can be employed to rapidly engineer immune cells and oncolytic viruses for cancer immunotherapeutic applications.
CRISPR-Cas9 can be used to rapidly engineer immune cells and oncolytic viruses for cancer immunotherapeutic applications.
CRISPR-Cas9 can be employed to rapidly engineer immune cells and oncolytic viruses for cancer immunotherapeutic applications.
CRISPR-Cas9 has clinical potential for discovering novel targets for cancer therapy and dissecting chemical-genetic interactions related to tumor drug response.
CRISPR-Cas9 has shown an unprecedented clinical potential to discover novel targets for cancer therapy and to dissect chemical-genetic interactions, providing insight into how tumours respond to drug treatment.
New variations of CRISPR/Cas9 had not yet been implemented in apicomplexans at the time of the review, and the technology's full potential remained unrealized pending integration of new variations and innovations.
we consider new variations of CRISPR/Cas9 that have yet to be implemented in apicomplexans... the full potential of this technology is yet to be realized as new variations and innovations are integrated into the field
CRISPR/Cas9 has been used for seminal genetic manipulations of Cryptosporidium species.
highlight its use for seminal genetic manipulations of Cryptosporidium spp.
The review documents implementation of CRISPR/Cas9 in apicomplexan parasites, especially Plasmodium spp. and Toxoplasma gondii.
documenting its implementation in apicomplexan parasites, especially Plasmodium spp. and Toxoplasma gondii
CRISPR/Cas9 has been used for whole-genome screening of gene knockout mutants in Toxoplasma gondii.
the recent use of CRISPR/Cas9 for whole genome screening of gene knockout mutants in T. gondii
CRISPR/Cas9 has been used for whole-genome screening of gene knockout mutants in Toxoplasma gondii.
the recent use of CRISPR/Cas9 for whole genome screening of gene knockout mutants in T. gondii
CRISPR/Cas9 has been used for whole-genome screening of gene knockout mutants in Toxoplasma gondii.
the recent use of CRISPR/Cas9 for whole genome screening of gene knockout mutants in T. gondii
CRISPR/Cas9 has been used for whole-genome screening of gene knockout mutants in Toxoplasma gondii.
the recent use of CRISPR/Cas9 for whole genome screening of gene knockout mutants in T. gondii
CRISPR/Cas9 has been used for whole-genome screening of gene knockout mutants in Toxoplasma gondii.
the recent use of CRISPR/Cas9 for whole genome screening of gene knockout mutants in T. gondii
CRISPR/Cas9 has been used for whole-genome screening of gene knockout mutants in Toxoplasma gondii.
the recent use of CRISPR/Cas9 for whole genome screening of gene knockout mutants in T. gondii
CRISPR/Cas9 has been used for whole-genome screening of gene knockout mutants in Toxoplasma gondii.
the recent use of CRISPR/Cas9 for whole genome screening of gene knockout mutants in T. gondii
CRISPR-Cas9 is presented as a potentially powerful tool for cancer therapy.
Because of its high efficiency and accuracy, the CRISPR-Cas9 genome editing technique has recently emerged as a potentially powerful tool in the arsenal of cancer therapy.
Important considerations and major challenges remain to be addressed before CRISPR/Cas9 can be clinically translated for cancer, a complex and polygenic disease.
In this review, we discuss important considerations for the use of CRISPR/Cas9 in therapeutic settings and major challenges that will need to be addressed prior to its clinical translation for a complex and polygenic disease such as cancer.
Targetable DNA-binding systems such as CRISPR/Cas9 and sensor-actuator devices enable precise control of complex cellular behaviors with high spatial and temporal resolution.
The invention of new research tools, including targetable DNA-binding systems such as CRISPR/Cas9 and sensor-actuator devices that can recognize and respond to diverse chemical, mechanical, and optical inputs, has enabled precise control of complex cellular behaviors at unprecedented spatial and temporal resolution.
Targetable DNA-binding systems such as CRISPR/Cas9 and sensor-actuator devices enable precise control of complex cellular behaviors with high spatial and temporal resolution.
The invention of new research tools, including targetable DNA-binding systems such as CRISPR/Cas9 and sensor-actuator devices that can recognize and respond to diverse chemical, mechanical, and optical inputs, has enabled precise control of complex cellular behaviors at unprecedented spatial and temporal resolution.
Targetable DNA-binding systems such as CRISPR/Cas9 and sensor-actuator devices enable precise control of complex cellular behaviors with high spatial and temporal resolution.
The invention of new research tools, including targetable DNA-binding systems such as CRISPR/Cas9 and sensor-actuator devices that can recognize and respond to diverse chemical, mechanical, and optical inputs, has enabled precise control of complex cellular behaviors at unprecedented spatial and temporal resolution.
Targetable DNA-binding systems such as CRISPR/Cas9 and sensor-actuator devices enable precise control of complex cellular behaviors with high spatial and temporal resolution.
The invention of new research tools, including targetable DNA-binding systems such as CRISPR/Cas9 and sensor-actuator devices that can recognize and respond to diverse chemical, mechanical, and optical inputs, has enabled precise control of complex cellular behaviors at unprecedented spatial and temporal resolution.
Targetable DNA-binding systems such as CRISPR/Cas9 and sensor-actuator devices enable precise control of complex cellular behaviors with high spatial and temporal resolution.
The invention of new research tools, including targetable DNA-binding systems such as CRISPR/Cas9 and sensor-actuator devices that can recognize and respond to diverse chemical, mechanical, and optical inputs, has enabled precise control of complex cellular behaviors at unprecedented spatial and temporal resolution.
Targetable DNA-binding systems such as CRISPR/Cas9 and sensor-actuator devices enable precise control of complex cellular behaviors with high spatial and temporal resolution.
The invention of new research tools, including targetable DNA-binding systems such as CRISPR/Cas9 and sensor-actuator devices that can recognize and respond to diverse chemical, mechanical, and optical inputs, has enabled precise control of complex cellular behaviors at unprecedented spatial and temporal resolution.
Targetable DNA-binding systems such as CRISPR/Cas9 and sensor-actuator devices enable precise control of complex cellular behaviors with high spatial and temporal resolution.
The invention of new research tools, including targetable DNA-binding systems such as CRISPR/Cas9 and sensor-actuator devices that can recognize and respond to diverse chemical, mechanical, and optical inputs, has enabled precise control of complex cellular behaviors at unprecedented spatial and temporal resolution.
Improvements in DNA sequencing and synthesis have expanded the set of genetic components available for programming mammalian cell biology.
Continued improvements in the capacity to sequence and synthesize DNA have rapidly increased our understanding of mechanisms of gene function and regulation on a genome-wide scale and have expanded the set of genetic components available for programming cell biology.
Improvements in DNA sequencing and synthesis have expanded the set of genetic components available for programming mammalian cell biology.
Continued improvements in the capacity to sequence and synthesize DNA have rapidly increased our understanding of mechanisms of gene function and regulation on a genome-wide scale and have expanded the set of genetic components available for programming cell biology.
Improvements in DNA sequencing and synthesis have expanded the set of genetic components available for programming mammalian cell biology.
Continued improvements in the capacity to sequence and synthesize DNA have rapidly increased our understanding of mechanisms of gene function and regulation on a genome-wide scale and have expanded the set of genetic components available for programming cell biology.
Improvements in DNA sequencing and synthesis have expanded the set of genetic components available for programming mammalian cell biology.
Continued improvements in the capacity to sequence and synthesize DNA have rapidly increased our understanding of mechanisms of gene function and regulation on a genome-wide scale and have expanded the set of genetic components available for programming cell biology.
Improvements in DNA sequencing and synthesis have expanded the set of genetic components available for programming mammalian cell biology.
Continued improvements in the capacity to sequence and synthesize DNA have rapidly increased our understanding of mechanisms of gene function and regulation on a genome-wide scale and have expanded the set of genetic components available for programming cell biology.
Improvements in DNA sequencing and synthesis have expanded the set of genetic components available for programming mammalian cell biology.
Continued improvements in the capacity to sequence and synthesize DNA have rapidly increased our understanding of mechanisms of gene function and regulation on a genome-wide scale and have expanded the set of genetic components available for programming cell biology.
Improvements in DNA sequencing and synthesis have expanded the set of genetic components available for programming mammalian cell biology.
Continued improvements in the capacity to sequence and synthesize DNA have rapidly increased our understanding of mechanisms of gene function and regulation on a genome-wide scale and have expanded the set of genetic components available for programming cell biology.
These tools were critical for extending synthetic biology techniques from prokaryotic and lower eukaryotic hosts to mammalian systems.
These tools have been critical for the expansion of synthetic biology techniques from prokaryotic and lower eukaryotic hosts to mammalian systems.
These tools were critical for extending synthetic biology techniques from prokaryotic and lower eukaryotic hosts to mammalian systems.
These tools have been critical for the expansion of synthetic biology techniques from prokaryotic and lower eukaryotic hosts to mammalian systems.
These tools were critical for extending synthetic biology techniques from prokaryotic and lower eukaryotic hosts to mammalian systems.
These tools have been critical for the expansion of synthetic biology techniques from prokaryotic and lower eukaryotic hosts to mammalian systems.
These tools were critical for extending synthetic biology techniques from prokaryotic and lower eukaryotic hosts to mammalian systems.
These tools have been critical for the expansion of synthetic biology techniques from prokaryotic and lower eukaryotic hosts to mammalian systems.
These tools were critical for extending synthetic biology techniques from prokaryotic and lower eukaryotic hosts to mammalian systems.
These tools have been critical for the expansion of synthetic biology techniques from prokaryotic and lower eukaryotic hosts to mammalian systems.
These tools were critical for extending synthetic biology techniques from prokaryotic and lower eukaryotic hosts to mammalian systems.
These tools have been critical for the expansion of synthetic biology techniques from prokaryotic and lower eukaryotic hosts to mammalian systems.
These tools were critical for extending synthetic biology techniques from prokaryotic and lower eukaryotic hosts to mammalian systems.
These tools have been critical for the expansion of synthetic biology techniques from prokaryotic and lower eukaryotic hosts to mammalian systems.
Progress in genome editing, epigenome editing, and programmable genetic circuits is expanding approaches to disease prevention, diagnosis, treatment, and personalized theranostic strategies.
Recent progress in the development of genome and epigenome editing tools and in the engineering of designer cells with programmable genetic circuits is expanding approaches to prevent, diagnose, and treat disease and to establish personalized theranostic strategies for next-generation medicines.
Progress in genome editing, epigenome editing, and programmable genetic circuits is expanding approaches to disease prevention, diagnosis, treatment, and personalized theranostic strategies.
Recent progress in the development of genome and epigenome editing tools and in the engineering of designer cells with programmable genetic circuits is expanding approaches to prevent, diagnose, and treat disease and to establish personalized theranostic strategies for next-generation medicines.
Progress in genome editing, epigenome editing, and programmable genetic circuits is expanding approaches to disease prevention, diagnosis, treatment, and personalized theranostic strategies.
Recent progress in the development of genome and epigenome editing tools and in the engineering of designer cells with programmable genetic circuits is expanding approaches to prevent, diagnose, and treat disease and to establish personalized theranostic strategies for next-generation medicines.
Progress in genome editing, epigenome editing, and programmable genetic circuits is expanding approaches to disease prevention, diagnosis, treatment, and personalized theranostic strategies.
Recent progress in the development of genome and epigenome editing tools and in the engineering of designer cells with programmable genetic circuits is expanding approaches to prevent, diagnose, and treat disease and to establish personalized theranostic strategies for next-generation medicines.
Progress in genome editing, epigenome editing, and programmable genetic circuits is expanding approaches to disease prevention, diagnosis, treatment, and personalized theranostic strategies.
Recent progress in the development of genome and epigenome editing tools and in the engineering of designer cells with programmable genetic circuits is expanding approaches to prevent, diagnose, and treat disease and to establish personalized theranostic strategies for next-generation medicines.
Progress in genome editing, epigenome editing, and programmable genetic circuits is expanding approaches to disease prevention, diagnosis, treatment, and personalized theranostic strategies.
Recent progress in the development of genome and epigenome editing tools and in the engineering of designer cells with programmable genetic circuits is expanding approaches to prevent, diagnose, and treat disease and to establish personalized theranostic strategies for next-generation medicines.
Progress in genome editing, epigenome editing, and programmable genetic circuits is expanding approaches to disease prevention, diagnosis, treatment, and personalized theranostic strategies.
Recent progress in the development of genome and epigenome editing tools and in the engineering of designer cells with programmable genetic circuits is expanding approaches to prevent, diagnose, and treat disease and to establish personalized theranostic strategies for next-generation medicines.
Possible applications of Cas9 in biomedical research and therapeutics are only beginning to be explored.
With all of these advances, we have just begun to explore the possible applications of Cas9 in biomedical research and therapeutics.
Possible applications of Cas9 in biomedical research and therapeutics are only beginning to be explored.
With all of these advances, we have just begun to explore the possible applications of Cas9 in biomedical research and therapeutics.
Possible applications of Cas9 in biomedical research and therapeutics are only beginning to be explored.
With all of these advances, we have just begun to explore the possible applications of Cas9 in biomedical research and therapeutics.
Possible applications of Cas9 in biomedical research and therapeutics are only beginning to be explored.
With all of these advances, we have just begun to explore the possible applications of Cas9 in biomedical research and therapeutics.
Possible applications of Cas9 in biomedical research and therapeutics are only beginning to be explored.
With all of these advances, we have just begun to explore the possible applications of Cas9 in biomedical research and therapeutics.
Possible applications of Cas9 in biomedical research and therapeutics are only beginning to be explored.
With all of these advances, we have just begun to explore the possible applications of Cas9 in biomedical research and therapeutics.
Possible applications of Cas9 in biomedical research and therapeutics are only beginning to be explored.
With all of these advances, we have just begun to explore the possible applications of Cas9 in biomedical research and therapeutics.
Cas9 is described as a powerful tool for engineering the genome in diverse organisms.
The Cas9 protein ... is emerging as a powerful tool for engineering the genome in diverse organisms.
Development of Cas9 as a tool made sequence-specific gene editing several magnitudes easier.
its development as a tool has made sequence-specific gene editing several magnitudes easier
Development of Cas9 as a tool made sequence-specific gene editing several magnitudes easier.
its development as a tool has made sequence-specific gene editing several magnitudes easier
Development of Cas9 as a tool made sequence-specific gene editing several magnitudes easier.
its development as a tool has made sequence-specific gene editing several magnitudes easier
Development of Cas9 as a tool made sequence-specific gene editing several magnitudes easier.
its development as a tool has made sequence-specific gene editing several magnitudes easier
Development of Cas9 as a tool made sequence-specific gene editing several magnitudes easier.
its development as a tool has made sequence-specific gene editing several magnitudes easier
Development of Cas9 as a tool made sequence-specific gene editing several magnitudes easier.
its development as a tool has made sequence-specific gene editing several magnitudes easier
Development of Cas9 as a tool made sequence-specific gene editing several magnitudes easier.
its development as a tool has made sequence-specific gene editing several magnitudes easier
Cas9 is an RNA-guided DNA endonuclease that can be reprogrammed to new target sites by changing the guide RNA sequence.
As an RNA-guided DNA endonuclease, Cas9 can be easily programmed to target new sites by altering its guide RNA sequence
CRISPR/Cas9 is described as a genome editing system that interrupts gene expression through cleavage of target DNA.
The bacterial Type II Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9 genome editing system is the latest method of interrupting gene expression through cleavage of target DNA.
siRNAs are described as a method for transient downregulation of target gene expression through the RNA interference pathway.
Short-interfering RNAs (siRNAs) are one method of transiently down regulating the expression of any target gene through the exploitation of the RNA interference pathway
siRNAs are described as a method for transient downregulation of target gene expression through the RNA interference pathway.
Short-interfering RNAs (siRNAs) are one method of transiently down regulating the expression of any target gene through the exploitation of the RNA interference pathway
siRNAs are described as a method for transient downregulation of target gene expression through the RNA interference pathway.
Short-interfering RNAs (siRNAs) are one method of transiently down regulating the expression of any target gene through the exploitation of the RNA interference pathway
siRNAs are described as a method for transient downregulation of target gene expression through the RNA interference pathway.
Short-interfering RNAs (siRNAs) are one method of transiently down regulating the expression of any target gene through the exploitation of the RNA interference pathway
siRNAs are described as a method for transient downregulation of target gene expression through the RNA interference pathway.
Short-interfering RNAs (siRNAs) are one method of transiently down regulating the expression of any target gene through the exploitation of the RNA interference pathway
siRNAs are described as a method for transient downregulation of target gene expression through the RNA interference pathway.
Short-interfering RNAs (siRNAs) are one method of transiently down regulating the expression of any target gene through the exploitation of the RNA interference pathway
siRNAs are described as a method for transient downregulation of target gene expression through the RNA interference pathway.
Short-interfering RNAs (siRNAs) are one method of transiently down regulating the expression of any target gene through the exploitation of the RNA interference pathway
TALENs are described as artificial systems that can be designed and constructed relatively quickly to bind practically anywhere in the genome and cleave double-stranded DNA, thereby interrupting expression of a target gene.
Transcription activator-like effector nucleases (TALENs) are artificial systems that can be designed and constructed relatively quickly to bind practically anywhere in the genome and cleave double stranded DNA, thus interrupting the expression of any given target gene
TALENs are described as artificial systems that can be designed and constructed relatively quickly to bind practically anywhere in the genome and cleave double-stranded DNA, thereby interrupting expression of a target gene.
Transcription activator-like effector nucleases (TALENs) are artificial systems that can be designed and constructed relatively quickly to bind practically anywhere in the genome and cleave double stranded DNA, thus interrupting the expression of any given target gene
TALENs are described as artificial systems that can be designed and constructed relatively quickly to bind practically anywhere in the genome and cleave double-stranded DNA, thereby interrupting expression of a target gene.
Transcription activator-like effector nucleases (TALENs) are artificial systems that can be designed and constructed relatively quickly to bind practically anywhere in the genome and cleave double stranded DNA, thus interrupting the expression of any given target gene
TALENs are described as artificial systems that can be designed and constructed relatively quickly to bind practically anywhere in the genome and cleave double-stranded DNA, thereby interrupting expression of a target gene.
Transcription activator-like effector nucleases (TALENs) are artificial systems that can be designed and constructed relatively quickly to bind practically anywhere in the genome and cleave double stranded DNA, thus interrupting the expression of any given target gene
TALENs are described as artificial systems that can be designed and constructed relatively quickly to bind practically anywhere in the genome and cleave double-stranded DNA, thereby interrupting expression of a target gene.
Transcription activator-like effector nucleases (TALENs) are artificial systems that can be designed and constructed relatively quickly to bind practically anywhere in the genome and cleave double stranded DNA, thus interrupting the expression of any given target gene
TALENs are described as artificial systems that can be designed and constructed relatively quickly to bind practically anywhere in the genome and cleave double-stranded DNA, thereby interrupting expression of a target gene.
Transcription activator-like effector nucleases (TALENs) are artificial systems that can be designed and constructed relatively quickly to bind practically anywhere in the genome and cleave double stranded DNA, thus interrupting the expression of any given target gene
TALENs are described as artificial systems that can be designed and constructed relatively quickly to bind practically anywhere in the genome and cleave double-stranded DNA, thereby interrupting expression of a target gene.
Transcription activator-like effector nucleases (TALENs) are artificial systems that can be designed and constructed relatively quickly to bind practically anywhere in the genome and cleave double stranded DNA, thus interrupting the expression of any given target gene
The abstract states that CRISPR/Cas9 has effectiveness at cleaving genomic DNA in mammalian cells in vitro and in vivo, exhibits specificity, and is relatively easy to construct in targeted forms.
Its effectiveness at cleaving genomic DNA in mammalian cells in vitro and in vivo [2, 3], the specificity that this system exhibits [4, 5] and the relative ease with which targeted systems can be constructed
This review focuses on four common gene-therapy-related modalities used to alter gene expression: siRNAs, TALENs, ZFNs, and CRISPR/Cas9.
Within this review we focus on 4 of the more common forms of gene therapy utilised to alter gene expression; siRNAs, TALENs, ZFNs and CRISPR/Cas9.
This review focuses on four common gene-therapy-related modalities used to alter gene expression: siRNAs, TALENs, ZFNs, and CRISPR/Cas9.
Within this review we focus on 4 of the more common forms of gene therapy utilised to alter gene expression; siRNAs, TALENs, ZFNs and CRISPR/Cas9.
This review focuses on four common gene-therapy-related modalities used to alter gene expression: siRNAs, TALENs, ZFNs, and CRISPR/Cas9.
Within this review we focus on 4 of the more common forms of gene therapy utilised to alter gene expression; siRNAs, TALENs, ZFNs and CRISPR/Cas9.
This review focuses on four common gene-therapy-related modalities used to alter gene expression: siRNAs, TALENs, ZFNs, and CRISPR/Cas9.
Within this review we focus on 4 of the more common forms of gene therapy utilised to alter gene expression; siRNAs, TALENs, ZFNs and CRISPR/Cas9.
This review focuses on four common gene-therapy-related modalities used to alter gene expression: siRNAs, TALENs, ZFNs, and CRISPR/Cas9.
Within this review we focus on 4 of the more common forms of gene therapy utilised to alter gene expression; siRNAs, TALENs, ZFNs and CRISPR/Cas9.
This review focuses on four common gene-therapy-related modalities used to alter gene expression: siRNAs, TALENs, ZFNs, and CRISPR/Cas9.
Within this review we focus on 4 of the more common forms of gene therapy utilised to alter gene expression; siRNAs, TALENs, ZFNs and CRISPR/Cas9.
This review focuses on four common gene-therapy-related modalities used to alter gene expression: siRNAs, TALENs, ZFNs, and CRISPR/Cas9.
Within this review we focus on 4 of the more common forms of gene therapy utilised to alter gene expression; siRNAs, TALENs, ZFNs and CRISPR/Cas9.
Approval Evidence
Studies have shown that the use of CRISPR-Cas9 to modulate key pathogenic pathways, including those governing inflammation, oxidative stress, and cell death, can prevent neuronal damage and improve neurological function.
Source:
This approach involves the use of programmable nucleases (CRISPR/Cas9, TALENs, ZFNs) that induce double-stranded DNA breaks at specific sites, allowing precise correction or targeted transgene integration.
Source:
This review also provides a comparative overview of prime editing and other gene-editing strategies for HbF modulation, such as CRISPR-Cas9 and Base editing.
Source:
Next-generation approaches, such as CRISPR-Cas9 and therapeutic aptamers, aim to inhibit viral replication, modulate oncogenic pathways, and enhance immune responses.
Source:
This work reviews the main progress achieved through transgenesis, induced mutagenesis, and precision gene editing, highlighting the role of tools such as RNAi, CRISPR/Cas9, and AlphaFold2-guided gene editing...
Source:
To address these limitations, combinatorial approaches, such as immune checkpoint inhibitors, cytokines, and advanced gene-editing tools like CRISPR/Cas9, are being actively explored.
Source:
Emerging gene delivery technologies, including viral vectors, transposons, CRISPR/Cas9, and RNA-based electroporation, are improving CAR-T production.
Source:
In contrast, the third generation Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR-associated protein 9 (CRISPR/Cas9), represents a significant breakthrough. It encompasses guided RNA (gRNA) and the Cas9 endonuclease which together target specific DNA sequences and induces double-strand breaks...
Source:
The upstream web research summary states that the anchor PMC text explicitly mentions CRISPR/Cas9 and frames CRISPR/Cas9 as a potential CNS therapeutic modality.
Source:
In the field of genetic engineering, precise gene editing tools, especially CRISPR/Cas9, have demonstrated considerable promise, although the application in enhancing microalgal protein production remains challenging and limited.
Source:
This review highlights the progression of organoid technologies, emphasizing the integration of genetic tools, including CRISPR-Cas9, prime editing, and lineage tracing.
Source:
Advances in gene-editing technologies, such as CRISPR/Cas9 and lentiviral vectors, have enabled the modification of HSCs to enhance their therapeutic potential... CRISPR/Cas9 offered precise editing but faced challenges with low homology-directed repair (HDR) efficiency in quiescent HSCs.
Source:
KSHV-associated malignancies lack virus-specific targeted treatments and current clinical outcomes remain suboptimal, especially in immunocompromised patients.
Source:
The review compares prime editing with CRISPR-Cas9 and Base editing as gene-editing strategies for HbF modulation.
This review also provides a comparative overview of prime editing and other gene-editing strategies for HbF modulation, such as CRISPR-Cas9 and Base editing.
Source:
Editing SWEET10a and SWEET10b allows modulation of the soybean oil-protein balance.
the editing of sugar transporters SWEET10a and SWEET10b allows the modulation of the oil-protein balance
Source:
Inactivation of genes related to antinutritional factors has reduced expression of phytate and protease inhibitors in soybean.
Simultaneously, the inactivation of genes related to antinutritional factors has significantly reduced the expression of compounds such as phytate and protease inhibitors.
Source:
Reviewed studies report that CRISPR-Cas9 modulation of inflammation, oxidative stress, and cell-death pathways can prevent neuronal damage and improve neurological function in ischemic stroke contexts.
Studies have shown that the use of CRISPR-Cas9 to modulate key pathogenic pathways, including those governing inflammation, oxidative stress, and cell death, can prevent neuronal damage and improve neurological function.
Source:
Programmable nucleases including CRISPR/Cas9, TALENs, and ZFNs induce double-stranded DNA breaks at specific sites, enabling precise correction or targeted transgene integration.
This approach involves the use of programmable nucleases (CRISPR/Cas9, TALENs, ZFNs) that induce double-stranded DNA breaks at specific sites, allowing precise correction or targeted transgene integration.
Source:
CRISPR-Cas9 is presented as a next-generation approach that aims to inhibit viral replication, modulate oncogenic pathways, and enhance immune responses in KSHV-associated disease.
Source:
Gene editing for hemophilia is presented as an emerging approach that aims to provide a permanent cure by precise correction of the mutated gene or targeted integration of coagulation factor cDNA for stable expression.
Gene editing for hemophilia is an emerging approach that aims to provide a permanent cure by editing the mutated gene precisely or targeted integration of coagulation factor cDNA into the host genome for stable expression.
Source:
RNAi, CRISPR/Cas9, and AlphaFold2-guided gene editing are used to modify genes involved in carbon and nitrogen metabolism and storage proteins in soybean.
This work reviews the main progress achieved through transgenesis, induced mutagenesis, and precision gene editing, highlighting the role of tools such as RNAi, CRISPR/Cas9, and AlphaFold2-guided gene editing in modifying genes involved in carbon and nitrogen metabolism and storage proteins.
Source:
The paper states that CRISPR/Cas9 in poultry has applications in disease resistance, productivity traits, in-ovo sexing, reproductive trait control, biopharming, and functional genomics.
CRISPR/Cas9 has diverse applications in poultry, including enhancing disease resistance to avian influenza and Marek's disease, improving productivity traits such as growth, feed efficiency, and egg-laying, and enabling early in-ovo sexing ... It also allows control of reproductive traits for breeding management, supports bio-pharming by producing therapeutic proteins or vaccines in eggs, and facilitates functional genomics...
Source:
CRISPR/Cas9 enables functional analysis of non-coding elements such as enhancers and insulators in addition to gene knockout.
Beyond gene knockout, CRISPR/Cas9 enables functional analysis of non-coding elements such as enhancers and insulators.
Source:
The review identifies long-term genetic stability, scalability, and off-target effects as challenges for genetically engineered tissues.
We address the field's challenges, including long-term genetic stability, scalability, and off-target effects, while also considering the ethical implications and evolving regulatory landscape of genetically engineered tissues.
Source:
Zinc finger nucleases and TALENs are limited by complex design and off-target effects relative to CRISPR/Cas9.
First and second generation tools, such as zinc finger nucleases and transcription activator-like effector nucleases (TALENs), are limited by complex design and off-target effects. In contrast, the third generation ... CRISPR/Cas9, represents a significant breakthrough.
Source:
When delivered via plasmid systems, Cas9 and gRNA are transiently expressed and degrade within 48-72 hours, leaving no permanent genetic footprint.
Delivered via plasmid systems, Cas9 and gRNA are transiently expressed and degrade within 48-72 h, leaving no permanent genetic footprint.
Source:
CRISPR/Cas9 provides precise editing in HSCs but is limited by low HDR efficiency in quiescent HSCs.
Source:
The explored improvement strategies aim to enhance CAR-T cell specificity, improve resistance to immunosuppressive signals, and optimize in vivo functionality.
Source:
Emerging gene editing approaches such as CRISPR/Cas9 are expanding treatment options and moving sickle cell disease gene therapy into clinical application.
Emerging gene editing approaches such as CRISPR/Cas9 are expanding treatment options, marking the transition of SCD gene therapy from theoretical concept to clinical application.
Source:
CRISPR/Cas9 has been used in vitro for gene correction or epigenetic activation, including SRY promoter demethylation in embryonic stem cells, and for targeted disruption of SOX9 enhancers in mice to model 46,XX testicular DSD.
CRISPR/Cas9 has been utilized to correct or epigenetically activate gene expression in vitro, such as SRY promoter demethylation in embryonic stem cells, and targeted disruption of SOX9 enhancers to model 46, XX testicular DSD in mice.
Source:
CRISPR/Cas9 targeting specificity is achieved through gRNA-DNA base pairing and Cas9 recognition of a protospacer adjacent motif.
Targeting specificity is achieved through gRNA-DNA base pairing and recognition of a protospacer adjacent motif by Cas9.
Source:
CRISPR/Cas9 uses gRNA and Cas9 to target specific DNA sequences and induce double-strand breaks that are often repaired by error-prone non-homologous end joining, frequently generating insertions or deletions that disrupt gene function.
It encompasses guided RNA (gRNA) and the Cas9 endonuclease which together target specific DNA sequences and induces double-strand breaks that are repaired via error-prone non-homologous end joining, frequently causing insertions or deletions that disrupt gene function.
Source:
Comparisons
Source-stated alternatives
The abstract mentions RNAi, AlphaFold2-guided gene editing, promoter engineering, Prime Editing, transgenesis, and induced mutagenesis as alternative or complementary approaches.; The abstract mentions therapeutic aptamers, conventional chemotherapy, antiretroviral therapy, and synthetic biology-based approaches as nearby therapeutic modalities.; The abstract explicitly mentions prime editing and Base editing as alternative gene-editing strategies in the same comparison.; The abstract contrasts CRISPR/Cas9 with other programmable nuclease classes including TALENs and ZFNs.; The review also discusses broader CRISPR-based systems and multiple delivery approaches rather than Cas9 alone. Viral vectors, nanocarriers, and extracellular vesicles are presented as alternative delivery harnesses.; The abstract contrasts CRISPR/Cas9-based gene editing with lentiviral vector-mediated gene insertion and with transplantation approaches.; The abstract directly contrasts CRISPR-Cas9 with TALENs and broader synthetic biology approaches. Base editing is also presented as an emerging related genetic engineering technology.; The review also discusses TALENs, ZFNs, and viral vector-mediated delivery systems as alternative or complementary gene-therapy approaches.; The abstract contrasts CRISPR/Cas9 with base editing and with non-viral delivery approaches such as electroporation.; The abstract names prime editing and lineage tracing as adjacent genetic approaches used alongside or instead of CRISPR-Cas9 in organoid studies.; The abstract directly contrasts CRISPR/Cas9 with random mutagenesis, which is described as proven effective across multiple strains.; The web research summary identifies Cpf1 as a nearby alternative RNA-guided nuclease discussed in related delivery literature.; The source contrasts CRISPR/Cas9 with zinc finger nucleases, TALENs, lentiviral vectors, and piggyBac transposition. ZFNs and TALENs are described as limited by complex design and off-target effects, while lentiviral and piggyBac methods are associated with exogenous gene expression.; Other production technologies named alongside it are viral vectors, transposons, and RNA-based electroporation.; Other combinatorial approaches named in the abstract include immune checkpoint inhibitors and cytokines.; The review contrasts CRISPR-enabled patient-independent or variant-induced models with patient-specific iPSC-CM models that inherit the donor genetic background.; The abstract contrasts gene editing with gene therapy by stating that gene editing, in addition to gene therapy, might be a promising strategy for PD therapy.; The abstract contrasts fly model generation with rodent model establishment, but does not name an alternative editing method.; The abstract contrasts CRISPR/Cas9 with other advancing modalities including gene therapy, stem cell therapy, and optogenetics.; The abstract names zinc finger proteins as another gene-editing technology.
Source:
The abstract mentions RNAi, AlphaFold2-guided gene editing, promoter engineering, Prime Editing, transgenesis, and induced mutagenesis as alternative or complementary approaches.
Source:
The abstract mentions therapeutic aptamers, conventional chemotherapy, antiretroviral therapy, and synthetic biology-based approaches as nearby therapeutic modalities.
Source:
The abstract explicitly mentions prime editing and Base editing as alternative gene-editing strategies in the same comparison.
Source:
The abstract contrasts CRISPR/Cas9 with other programmable nuclease classes including TALENs and ZFNs.
Source:
The review also discusses broader CRISPR-based systems and multiple delivery approaches rather than Cas9 alone. Viral vectors, nanocarriers, and extracellular vesicles are presented as alternative delivery harnesses.
Source:
The abstract contrasts CRISPR/Cas9-based gene editing with lentiviral vector-mediated gene insertion and with transplantation approaches.
Source:
The abstract directly contrasts CRISPR-Cas9 with TALENs and broader synthetic biology approaches. Base editing is also presented as an emerging related genetic engineering technology.
Source:
The review also discusses TALENs, ZFNs, and viral vector-mediated delivery systems as alternative or complementary gene-therapy approaches.
Source:
The abstract contrasts CRISPR/Cas9 with base editing and with non-viral delivery approaches such as electroporation.
Source:
The abstract names prime editing and lineage tracing as adjacent genetic approaches used alongside or instead of CRISPR-Cas9 in organoid studies.
Source:
The abstract directly contrasts CRISPR/Cas9 with random mutagenesis, which is described as proven effective across multiple strains.
Source:
The web research summary identifies Cpf1 as a nearby alternative RNA-guided nuclease discussed in related delivery literature.
Source:
The source contrasts CRISPR/Cas9 with zinc finger nucleases, TALENs, lentiviral vectors, and piggyBac transposition. ZFNs and TALENs are described as limited by complex design and off-target effects, while lentiviral and piggyBac methods are associated with exogenous gene expression.
Source:
Other production technologies named alongside it are viral vectors, transposons, and RNA-based electroporation.
Source:
Other combinatorial approaches named in the abstract include immune checkpoint inhibitors and cytokines.
Source:
The review contrasts CRISPR-enabled patient-independent or variant-induced models with patient-specific iPSC-CM models that inherit the donor genetic background.
Source:
The abstract contrasts gene editing with gene therapy by stating that gene editing, in addition to gene therapy, might be a promising strategy for PD therapy.
Source:
The abstract contrasts fly model generation with rodent model establishment, but does not name an alternative editing method.
Source:
The abstract contrasts CRISPR/Cas9 with other advancing modalities including gene therapy, stem cell therapy, and optogenetics.
Source:
The abstract names zinc finger proteins as another gene-editing technology.
Source-backed strengths
The evidence attributes high efficiency and accuracy to the CRISPR-Cas9 genome editing technique and notes many advantages without detailing all of them. It was engineered for mammalian cell gene editing and is described as broadening gene therapy toward site-specific genome modification.
Source:
The ease of design, low cytotoxicity, and increased efficiency have made CRISPR/Cas9 and its related systems the designer nucleases of choice for many.
Source:
While these approaches provided sequence-specific gene-editing capacity, the laborious process of designing and synthesizing recombinant nucleases to recognize a specific target sequence, combined with limited target choices and poor editing efficiency, ultimately minimized the broad utility of these systems.
Source:
its development as a tool has made sequence-specific gene editing several magnitudes easier
Source:
Its effectiveness at cleaving genomic DNA in mammalian cells in vitro and in vivo [2, 3], the specificity that this system exhibits [4, 5] and the relative ease with which targeted systems can be constructed
Compared with Adeno-associated virus
The review also discusses broader CRISPR-based systems and multiple delivery approaches rather than Cas9 alone. Viral vectors, nanocarriers, and extracellular vesicles are presented as alternative delivery harnesses.; Other production technologies named alongside it are viral vectors, transposons, and RNA-based electroporation.
Shared frame: source-stated alternative in extracted literature
Strengths here: presented as a precision gene-editing tool for soybean grain-quality engineering; presented as a next-generation targeted approach; programmable nuclease platform for site-specific DNA cleavage.
Relative tradeoffs: specific implementation limits are not described in the abstract; persistent challenges in targeted delivery; persistent challenges in safe delivery.
Source:
The review also discusses broader CRISPR-based systems and multiple delivery approaches rather than Cas9 alone. Viral vectors, nanocarriers, and extracellular vesicles are presented as alternative delivery harnesses.
Source:
Other production technologies named alongside it are viral vectors, transposons, and RNA-based electroporation.
Compared with base editing
The abstract explicitly mentions prime editing and Base editing as alternative gene-editing strategies in the same comparison.; The abstract directly contrasts CRISPR-Cas9 with TALENs and broader synthetic biology approaches. Base editing is also presented as an emerging related genetic engineering technology.; The abstract contrasts CRISPR/Cas9 with base editing and with non-viral delivery approaches such as electroporation.
Shared frame: source-stated alternative in extracted literature
Strengths here: presented as a precision gene-editing tool for soybean grain-quality engineering; presented as a next-generation targeted approach; programmable nuclease platform for site-specific DNA cleavage.
Relative tradeoffs: specific implementation limits are not described in the abstract; persistent challenges in targeted delivery; persistent challenges in safe delivery.
Source:
The abstract explicitly mentions prime editing and Base editing as alternative gene-editing strategies in the same comparison.
Source:
The abstract directly contrasts CRISPR-Cas9 with TALENs and broader synthetic biology approaches. Base editing is also presented as an emerging related genetic engineering technology.
Source:
The abstract contrasts CRISPR/Cas9 with base editing and with non-viral delivery approaches such as electroporation.
Compared with CRISPR/Cas9 system
The abstract contrasts CRISPR/Cas9 with other programmable nuclease classes including TALENs and ZFNs.; The review also discusses broader CRISPR-based systems and multiple delivery approaches rather than Cas9 alone. Viral vectors, nanocarriers, and extracellular vesicles are presented as alternative delivery harnesses.; The abstract contrasts CRISPR/Cas9-based gene editing with lentiviral vector-mediated gene insertion and with transplantation approaches.; The abstract directly contrasts CRISPR-Cas9 with TALENs and broader synthetic biology approaches. Base editing is also presented as an emerging related genetic engineering technology.; The abstract contrasts CRISPR/Cas9 with base editing and with non-viral delivery approaches such as electroporation.; The abstract names prime editing and lineage tracing as adjacent genetic approaches used alongside or instead of CRISPR-Cas9 in organoid studies.; The abstract directly contrasts CRISPR/Cas9 with random mutagenesis, which is described as proven effective across multiple strains.; The source contrasts CRISPR/Cas9 with zinc finger nucleases, TALENs, lentiviral vectors, and piggyBac transposition. ZFNs and TALENs are described as limited by complex design and off-target effects, while lentiviral and piggyBac methods are associated with exogenous gene expression.; The review contrasts CRISPR-enabled patient-independent or variant-induced models with patient-specific iPSC-CM models that inherit the donor genetic background.; The abstract contrasts CRISPR/Cas9 with other advancing modalities including gene therapy, stem cell therapy, and optogenetics.
Shared frame: source-stated alternative in extracted literature
Strengths here: presented as a precision gene-editing tool for soybean grain-quality engineering; presented as a next-generation targeted approach; programmable nuclease platform for site-specific DNA cleavage.
Relative tradeoffs: specific implementation limits are not described in the abstract; persistent challenges in targeted delivery; persistent challenges in safe delivery.
Source:
The abstract contrasts CRISPR/Cas9 with other programmable nuclease classes including TALENs and ZFNs.
Source:
The review also discusses broader CRISPR-based systems and multiple delivery approaches rather than Cas9 alone. Viral vectors, nanocarriers, and extracellular vesicles are presented as alternative delivery harnesses.
Source:
The abstract contrasts CRISPR/Cas9-based gene editing with lentiviral vector-mediated gene insertion and with transplantation approaches.
Source:
The abstract directly contrasts CRISPR-Cas9 with TALENs and broader synthetic biology approaches. Base editing is also presented as an emerging related genetic engineering technology.
Source:
The abstract contrasts CRISPR/Cas9 with base editing and with non-viral delivery approaches such as electroporation.
Source:
The abstract names prime editing and lineage tracing as adjacent genetic approaches used alongside or instead of CRISPR-Cas9 in organoid studies.
Source:
The abstract directly contrasts CRISPR/Cas9 with random mutagenesis, which is described as proven effective across multiple strains.
Source:
The source contrasts CRISPR/Cas9 with zinc finger nucleases, TALENs, lentiviral vectors, and piggyBac transposition. ZFNs and TALENs are described as limited by complex design and off-target effects, while lentiviral and piggyBac methods are associated with exogenous gene expression.
Source:
The review contrasts CRISPR-enabled patient-independent or variant-induced models with patient-specific iPSC-CM models that inherit the donor genetic background.
Source:
The abstract contrasts CRISPR/Cas9 with other advancing modalities including gene therapy, stem cell therapy, and optogenetics.
Compared with Exosomes
The review also discusses broader CRISPR-based systems and multiple delivery approaches rather than Cas9 alone. Viral vectors, nanocarriers, and extracellular vesicles are presented as alternative delivery harnesses.
Shared frame: source-stated alternative in extracted literature
Strengths here: presented as a precision gene-editing tool for soybean grain-quality engineering; presented as a next-generation targeted approach; programmable nuclease platform for site-specific DNA cleavage.
Relative tradeoffs: specific implementation limits are not described in the abstract; persistent challenges in targeted delivery; persistent challenges in safe delivery.
Source:
The review also discusses broader CRISPR-based systems and multiple delivery approaches rather than Cas9 alone. Viral vectors, nanocarriers, and extracellular vesicles are presented as alternative delivery harnesses.
Compared with extracellular vesicles
The review also discusses broader CRISPR-based systems and multiple delivery approaches rather than Cas9 alone. Viral vectors, nanocarriers, and extracellular vesicles are presented as alternative delivery harnesses.
Shared frame: source-stated alternative in extracted literature
Strengths here: presented as a precision gene-editing tool for soybean grain-quality engineering; presented as a next-generation targeted approach; programmable nuclease platform for site-specific DNA cleavage.
Relative tradeoffs: specific implementation limits are not described in the abstract; persistent challenges in targeted delivery; persistent challenges in safe delivery.
Source:
The review also discusses broader CRISPR-based systems and multiple delivery approaches rather than Cas9 alone. Viral vectors, nanocarriers, and extracellular vesicles are presented as alternative delivery harnesses.
Compared with gene therapy
The abstract contrasts gene editing with gene therapy by stating that gene editing, in addition to gene therapy, might be a promising strategy for PD therapy.; The abstract contrasts CRISPR/Cas9 with other advancing modalities including gene therapy, stem cell therapy, and optogenetics.
Shared frame: source-stated alternative in extracted literature
Strengths here: presented as a precision gene-editing tool for soybean grain-quality engineering; presented as a next-generation targeted approach; programmable nuclease platform for site-specific DNA cleavage.
Relative tradeoffs: specific implementation limits are not described in the abstract; persistent challenges in targeted delivery; persistent challenges in safe delivery.
Source:
The abstract contrasts gene editing with gene therapy by stating that gene editing, in addition to gene therapy, might be a promising strategy for PD therapy.
Source:
The abstract contrasts CRISPR/Cas9 with other advancing modalities including gene therapy, stem cell therapy, and optogenetics.
Compared with lipid nanoparticles
The review also discusses broader CRISPR-based systems and multiple delivery approaches rather than Cas9 alone. Viral vectors, nanocarriers, and extracellular vesicles are presented as alternative delivery harnesses.
Shared frame: source-stated alternative in extracted literature
Strengths here: presented as a precision gene-editing tool for soybean grain-quality engineering; presented as a next-generation targeted approach; programmable nuclease platform for site-specific DNA cleavage.
Relative tradeoffs: specific implementation limits are not described in the abstract; persistent challenges in targeted delivery; persistent challenges in safe delivery.
Source:
The review also discusses broader CRISPR-based systems and multiple delivery approaches rather than Cas9 alone. Viral vectors, nanocarriers, and extracellular vesicles are presented as alternative delivery harnesses.
Compared with optogenetic functional interrogation
The abstract contrasts CRISPR/Cas9 with other advancing modalities including gene therapy, stem cell therapy, and optogenetics.
Shared frame: source-stated alternative in extracted literature
Strengths here: presented as a precision gene-editing tool for soybean grain-quality engineering; presented as a next-generation targeted approach; programmable nuclease platform for site-specific DNA cleavage.
Relative tradeoffs: specific implementation limits are not described in the abstract; persistent challenges in targeted delivery; persistent challenges in safe delivery.
Source:
The abstract contrasts CRISPR/Cas9 with other advancing modalities including gene therapy, stem cell therapy, and optogenetics.
Compared with optogenetic membrane potential perturbation
The abstract contrasts CRISPR/Cas9 with other advancing modalities including gene therapy, stem cell therapy, and optogenetics.
Shared frame: source-stated alternative in extracted literature
Strengths here: presented as a precision gene-editing tool for soybean grain-quality engineering; presented as a next-generation targeted approach; programmable nuclease platform for site-specific DNA cleavage.
Relative tradeoffs: specific implementation limits are not described in the abstract; persistent challenges in targeted delivery; persistent challenges in safe delivery.
Source:
The abstract contrasts CRISPR/Cas9 with other advancing modalities including gene therapy, stem cell therapy, and optogenetics.
Compared with polymeric vesicles
The review also discusses broader CRISPR-based systems and multiple delivery approaches rather than Cas9 alone. Viral vectors, nanocarriers, and extracellular vesicles are presented as alternative delivery harnesses.
Shared frame: source-stated alternative in extracted literature
Strengths here: presented as a precision gene-editing tool for soybean grain-quality engineering; presented as a next-generation targeted approach; programmable nuclease platform for site-specific DNA cleavage.
Relative tradeoffs: specific implementation limits are not described in the abstract; persistent challenges in targeted delivery; persistent challenges in safe delivery.
Source:
The review also discusses broader CRISPR-based systems and multiple delivery approaches rather than Cas9 alone. Viral vectors, nanocarriers, and extracellular vesicles are presented as alternative delivery harnesses.
Compared with prime-editing
The abstract mentions RNAi, AlphaFold2-guided gene editing, promoter engineering, Prime Editing, transgenesis, and induced mutagenesis as alternative or complementary approaches.; The abstract explicitly mentions prime editing and Base editing as alternative gene-editing strategies in the same comparison.; The abstract names prime editing and lineage tracing as adjacent genetic approaches used alongside or instead of CRISPR-Cas9 in organoid studies.
Shared frame: source-stated alternative in extracted literature
Strengths here: presented as a precision gene-editing tool for soybean grain-quality engineering; presented as a next-generation targeted approach; programmable nuclease platform for site-specific DNA cleavage.
Relative tradeoffs: specific implementation limits are not described in the abstract; persistent challenges in targeted delivery; persistent challenges in safe delivery.
Source:
The abstract mentions RNAi, AlphaFold2-guided gene editing, promoter engineering, Prime Editing, transgenesis, and induced mutagenesis as alternative or complementary approaches.
Source:
The abstract explicitly mentions prime editing and Base editing as alternative gene-editing strategies in the same comparison.
Source:
The abstract names prime editing and lineage tracing as adjacent genetic approaches used alongside or instead of CRISPR-Cas9 in organoid studies.
Compared with transcription activator-like effector nucleases
The abstract contrasts CRISPR/Cas9 with other programmable nuclease classes including TALENs and ZFNs.; The abstract directly contrasts CRISPR-Cas9 with TALENs and broader synthetic biology approaches. Base editing is also presented as an emerging related genetic engineering technology.; The review also discusses TALENs, ZFNs, and viral vector-mediated delivery systems as alternative or complementary gene-therapy approaches.; The source contrasts CRISPR/Cas9 with zinc finger nucleases, TALENs, lentiviral vectors, and piggyBac transposition. ZFNs and TALENs are described as limited by complex design and off-target effects, while lentiviral and piggyBac methods are associated with exogenous gene expression.
Shared frame: source-stated alternative in extracted literature
Strengths here: presented as a precision gene-editing tool for soybean grain-quality engineering; presented as a next-generation targeted approach; programmable nuclease platform for site-specific DNA cleavage.
Relative tradeoffs: specific implementation limits are not described in the abstract; persistent challenges in targeted delivery; persistent challenges in safe delivery.
Source:
The abstract contrasts CRISPR/Cas9 with other programmable nuclease classes including TALENs and ZFNs.
Source:
The abstract directly contrasts CRISPR-Cas9 with TALENs and broader synthetic biology approaches. Base editing is also presented as an emerging related genetic engineering technology.
Source:
The review also discusses TALENs, ZFNs, and viral vector-mediated delivery systems as alternative or complementary gene-therapy approaches.
Source:
The source contrasts CRISPR/Cas9 with zinc finger nucleases, TALENs, lentiviral vectors, and piggyBac transposition. ZFNs and TALENs are described as limited by complex design and off-target effects, while lentiviral and piggyBac methods are associated with exogenous gene expression.
Compared with zinc finger nucleases
The abstract contrasts CRISPR/Cas9 with other programmable nuclease classes including TALENs and ZFNs.; The review also discusses TALENs, ZFNs, and viral vector-mediated delivery systems as alternative or complementary gene-therapy approaches.; The source contrasts CRISPR/Cas9 with zinc finger nucleases, TALENs, lentiviral vectors, and piggyBac transposition. ZFNs and TALENs are described as limited by complex design and off-target effects, while lentiviral and piggyBac methods are associated with exogenous gene expression.
Shared frame: source-stated alternative in extracted literature
Strengths here: presented as a precision gene-editing tool for soybean grain-quality engineering; presented as a next-generation targeted approach; programmable nuclease platform for site-specific DNA cleavage.
Relative tradeoffs: specific implementation limits are not described in the abstract; persistent challenges in targeted delivery; persistent challenges in safe delivery.
Source:
The abstract contrasts CRISPR/Cas9 with other programmable nuclease classes including TALENs and ZFNs.
Source:
The review also discusses TALENs, ZFNs, and viral vector-mediated delivery systems as alternative or complementary gene-therapy approaches.
Source:
The source contrasts CRISPR/Cas9 with zinc finger nucleases, TALENs, lentiviral vectors, and piggyBac transposition. ZFNs and TALENs are described as limited by complex design and off-target effects, while lentiviral and piggyBac methods are associated with exogenous gene expression.
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