Toolkit/antisense oligonucleotides

antisense oligonucleotides

RNA Element·Research·Since 2024

Also known as: ASOs

Taxonomy: Mechanism Branch / Component. Workflows sit above the mechanism and technique branches rather than replacing them.

Summary

Gene therapies such as antisense oligonucleotides (ASOs) ... have played crucial roles in discovering and validating new pain targets.

Usefulness & Problems

Why this is useful

ASOs are presented as a major class of RNA therapeutics within RNA-targeted therapy. The review places them among strategies used for gene silencing and other RNA-directed applications.; gene silencing; RNA-targeted therapy; Antisense oligonucleotides are presented as a therapeutic component class for modulating RNA targets in TNBC-related ncRNA therapy.; therapeutic modulation of ncRNA-related targets in TNBC; ASOs are presented as a gene-therapy strategy used to modulate pain-relevant molecular targets. The review frames them as tools for discovering and validating new pain targets.; gene-based modulation of pain-relevant targets; target discovery and validation in chronic pain

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ASOs are presented as a major class of RNA therapeutics within RNA-targeted therapy. The review places them among strategies used for gene silencing and other RNA-directed applications.

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gene silencing

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RNA-targeted therapy

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Antisense oligonucleotides are presented as a therapeutic component class for modulating RNA targets in TNBC-related ncRNA therapy.

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therapeutic modulation of ncRNA-related targets in TNBC

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ASOs are presented as a gene-therapy strategy used to modulate pain-relevant molecular targets. The review frames them as tools for discovering and validating new pain targets.

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gene-based modulation of pain-relevant targets

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target discovery and validation in chronic pain

Problem solved

ASOs help address disease by targeting RNA rather than proteins, expanding therapeutic intervention at the RNA-regulatory level.; providing an RNA-based therapeutic modality for disease treatment; They offer a direct way to intervene at the RNA level rather than through protein-targeted drugs.; providing a direct oligonucleotide-based route to modulate RNA targets; They help address molecular targets that remain difficult to manipulate with traditional methods in chronic pain research and therapy.; helps address pain targets that are difficult to target with traditional methods

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ASOs help address disease by targeting RNA rather than proteins, expanding therapeutic intervention at the RNA-regulatory level.

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providing an RNA-based therapeutic modality for disease treatment

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They offer a direct way to intervene at the RNA level rather than through protein-targeted drugs.

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providing a direct oligonucleotide-based route to modulate RNA targets

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They help address molecular targets that remain difficult to manipulate with traditional methods in chronic pain research and therapy.

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helps address pain targets that are difficult to target with traditional methods

Problem links

helps address pain targets that are difficult to target with traditional methods

Literature

They help address molecular targets that remain difficult to manipulate with traditional methods in chronic pain research and therapy.

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They help address molecular targets that remain difficult to manipulate with traditional methods in chronic pain research and therapy.

providing a direct oligonucleotide-based route to modulate RNA targets

Literature

They offer a direct way to intervene at the RNA level rather than through protein-targeted drugs.

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They offer a direct way to intervene at the RNA level rather than through protein-targeted drugs.

providing an RNA-based therapeutic modality for disease treatment

Literature

ASOs help address disease by targeting RNA rather than proteins, expanding therapeutic intervention at the RNA-regulatory level.

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ASOs help address disease by targeting RNA rather than proteins, expanding therapeutic intervention at the RNA-regulatory level.

Taxonomy & Function

Primary hierarchy

Mechanism Branch

Component: A low-level RNA part used inside a larger architecture that realizes a mechanism.

Mechanisms

No mechanism tags yet.

Techniques

No technique tags yet.

Target processes

editingrecombination

Input: Light

Implementation Constraints

cofactor dependency: cofactor requirement unknownencoding mode: genetically encodedimplementation constraint: context specific validationimplementation constraint: payload burdenimplementation constraint: spectral hardware requirementoperating role: sensor

The abstract links progress in RNA therapeutics broadly to advances in delivery technologies, chemical modification, and related enabling methods. It does not specify a single ASO delivery platform in the abstract.; requires sufficient in vivo stability; requires effective delivery; They require target-specific oligonucleotide design and, in practice, a delivery context, though the supplied payload does not detail exact chemistries or carriers.; requires sequence-specific oligonucleotide design against the intended RNA target; likely depends on a delivery strategy, but the anchor review text was not provided to specify this; Use requires a delivery approach to the relevant pain-associated cells or tissues. The abstract does not specify chemistry, route, or formulation details.; requires delivery to target cells relevant to pain

The abstract states that translational barriers remain, including in vivo stability, delivery efficiency, and immune activation.; broader clinical application is limited by translational barriers; common challenges include in vivo stability, delivery efficiency, and immune activation; The current evidence does not identify which ASO formats, targets, or delivery systems are most successful in the reviewed studies.; the provided payload does not specify exact target classes, chemistries, or delivery dependencies from the anchor review; The abstract does not show that ASOs broadly solve clinical pain treatment on their own, and it notes that pain-focused gene therapy trials remain uncommon.

Validation

Cell-freeBacteriaMammalianMouseHumanTherapeuticIndep. Replication

Supporting Sources

Ranked Claims

Claim 1delivery modality recurrencesupports2026Source 2needs review

Exosomes, nanoparticles, liposomes, aptamer-siRNA conjugates, and antisense oligonucleotides are presented in the supplied summary as recurring or relevant component classes for TNBC ncRNA therapeutics.

Claim 2review summarysupports2024Source 1needs review

Gene-therapy modalities including ASOs, RNAi, CRISPR, and virus-based delivery systems have played crucial roles in discovering and validating new pain targets.

Claim 3scope of reviewsupports2024Source 1needs review

The review covers ASOs, siRNAs, optogenetics, chemogenetics, CRISPR, and their delivery methods targeting primary sensory neurons and non-neuronal cells including glia and chondrocytes.

Claim 4translational landscapesupports2024Source 1needs review

Although gene therapy-based clinical trials have increased, trials focused on pain as the primary outcome remain uncommon.

Approval Evidence

3 sources9 linked approval claimsfirst-pass slug antisense-oligonucleotides
The supplied web research summary lists antisense oligonucleotides as an explicit therapeutic component class relevant to the anchor review topic.

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Advances in high-throughput sequencing, structural biology, and delivery technologies have accelerated the development of diverse RNA therapeutics, including antisense oligonucleotides (ASOs).

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Gene therapies such as antisense oligonucleotides (ASOs) ... have played crucial roles in discovering and validating new pain targets.

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application scopesupports

RNA-based strategies are applied to gene silencing, editing, protein replacement, immune activation, and targeted drug delivery.

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delivery modality recurrencesupports

Exosomes, nanoparticles, liposomes, aptamer-siRNA conjugates, and antisense oligonucleotides are presented in the supplied summary as recurring or relevant component classes for TNBC ncRNA therapeutics.

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disease emphasissupports

ASOs and siRNAs receive special emphasis for neurological, metabolic, and infectious diseases.

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field shiftsupports

RNA-targeted therapy is shifting molecular medicine from a protein-centric view toward an RNA-regulatory network paradigm.

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modality coveragesupports

Diverse RNA therapeutics include ASOs, siRNA, miRNA modulators, mRNA therapeutics, aptamers, shRNA, and CRISPR/Cas-guided single-guide RNAs.

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translational barriersupports

Common challenges for RNA therapeutics include in vivo stability, delivery efficiency, and immune activation.

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review summarysupports

Gene-therapy modalities including ASOs, RNAi, CRISPR, and virus-based delivery systems have played crucial roles in discovering and validating new pain targets.

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scope of reviewsupports

The review covers ASOs, siRNAs, optogenetics, chemogenetics, CRISPR, and their delivery methods targeting primary sensory neurons and non-neuronal cells including glia and chondrocytes.

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translational landscapesupports

Although gene therapy-based clinical trials have increased, trials focused on pain as the primary outcome remain uncommon.

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Comparisons

Source-stated alternatives

The review compares ASOs with other RNA modalities including siRNA, miRNA modulators, mRNA therapeutics, aptamers, shRNA, and CRISPR/Cas-guided single-guide RNAs.; The supplied summary points to exosomes, nanoparticles, liposomes, and aptamer-siRNA systems as adjacent approaches.; The review contrasts ASOs with RNAi, siRNAs, CRISPR, optogenetics, chemogenetics, and virus-based delivery systems.

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The review compares ASOs with other RNA modalities including siRNA, miRNA modulators, mRNA therapeutics, aptamers, shRNA, and CRISPR/Cas-guided single-guide RNAs.

Source:

The supplied summary points to exosomes, nanoparticles, liposomes, and aptamer-siRNA systems as adjacent approaches.

Source:

The review contrasts ASOs with RNAi, siRNAs, CRISPR, optogenetics, chemogenetics, and virus-based delivery systems.

Source-backed strengths

highlighted as a major RNA therapeutic modality; given special emphasis for neurological, metabolic, and infectious diseases; explicitly named as a therapeutic component class in the supplied summary; explicitly described as playing a crucial role in discovering and validating new pain targets

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highlighted as a major RNA therapeutic modality

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given special emphasis for neurological, metabolic, and infectious diseases

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explicitly named as a therapeutic component class in the supplied summary

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explicitly described as playing a crucial role in discovering and validating new pain targets

The review compares ASOs with other RNA modalities including siRNA, miRNA modulators, mRNA therapeutics, aptamers, shRNA, and CRISPR/Cas-guided single-guide RNAs.; The review contrasts ASOs with RNAi, siRNAs, CRISPR, optogenetics, chemogenetics, and virus-based delivery systems.

Shared frame: source-stated alternative in extracted literature

Strengths here: highlighted as a major RNA therapeutic modality; given special emphasis for neurological, metabolic, and infectious diseases; explicitly named as a therapeutic component class in the supplied summary.

Relative tradeoffs: broader clinical application is limited by translational barriers; common challenges include in vivo stability, delivery efficiency, and immune activation; the provided payload does not specify exact target classes, chemistries, or delivery dependencies from the anchor review.

Source:

The review compares ASOs with other RNA modalities including siRNA, miRNA modulators, mRNA therapeutics, aptamers, shRNA, and CRISPR/Cas-guided single-guide RNAs.

Source:

The review contrasts ASOs with RNAi, siRNAs, CRISPR, optogenetics, chemogenetics, and virus-based delivery systems.

The review compares ASOs with other RNA modalities including siRNA, miRNA modulators, mRNA therapeutics, aptamers, shRNA, and CRISPR/Cas-guided single-guide RNAs.; The review contrasts ASOs with RNAi, siRNAs, CRISPR, optogenetics, chemogenetics, and virus-based delivery systems.

Shared frame: source-stated alternative in extracted literature

Strengths here: highlighted as a major RNA therapeutic modality; given special emphasis for neurological, metabolic, and infectious diseases; explicitly named as a therapeutic component class in the supplied summary.

Relative tradeoffs: broader clinical application is limited by translational barriers; common challenges include in vivo stability, delivery efficiency, and immune activation; the provided payload does not specify exact target classes, chemistries, or delivery dependencies from the anchor review.

Source:

The review compares ASOs with other RNA modalities including siRNA, miRNA modulators, mRNA therapeutics, aptamers, shRNA, and CRISPR/Cas-guided single-guide RNAs.

Source:

The review contrasts ASOs with RNAi, siRNAs, CRISPR, optogenetics, chemogenetics, and virus-based delivery systems.

The review compares ASOs with other RNA modalities including siRNA, miRNA modulators, mRNA therapeutics, aptamers, shRNA, and CRISPR/Cas-guided single-guide RNAs.

Shared frame: source-stated alternative in extracted literature

Strengths here: highlighted as a major RNA therapeutic modality; given special emphasis for neurological, metabolic, and infectious diseases; explicitly named as a therapeutic component class in the supplied summary.

Relative tradeoffs: broader clinical application is limited by translational barriers; common challenges include in vivo stability, delivery efficiency, and immune activation; the provided payload does not specify exact target classes, chemistries, or delivery dependencies from the anchor review.

Source:

The review compares ASOs with other RNA modalities including siRNA, miRNA modulators, mRNA therapeutics, aptamers, shRNA, and CRISPR/Cas-guided single-guide RNAs.

Compared with chemogenetics

The review contrasts ASOs with RNAi, siRNAs, CRISPR, optogenetics, chemogenetics, and virus-based delivery systems.

Shared frame: source-stated alternative in extracted literature

Strengths here: highlighted as a major RNA therapeutic modality; given special emphasis for neurological, metabolic, and infectious diseases; explicitly named as a therapeutic component class in the supplied summary.

Relative tradeoffs: broader clinical application is limited by translational barriers; common challenges include in vivo stability, delivery efficiency, and immune activation; the provided payload does not specify exact target classes, chemistries, or delivery dependencies from the anchor review.

Source:

The review contrasts ASOs with RNAi, siRNAs, CRISPR, optogenetics, chemogenetics, and virus-based delivery systems.

Compared with CRISPR/Cas9

The review compares ASOs with other RNA modalities including siRNA, miRNA modulators, mRNA therapeutics, aptamers, shRNA, and CRISPR/Cas-guided single-guide RNAs.; The review contrasts ASOs with RNAi, siRNAs, CRISPR, optogenetics, chemogenetics, and virus-based delivery systems.

Shared frame: source-stated alternative in extracted literature

Strengths here: highlighted as a major RNA therapeutic modality; given special emphasis for neurological, metabolic, and infectious diseases; explicitly named as a therapeutic component class in the supplied summary.

Relative tradeoffs: broader clinical application is limited by translational barriers; common challenges include in vivo stability, delivery efficiency, and immune activation; the provided payload does not specify exact target classes, chemistries, or delivery dependencies from the anchor review.

Source:

The review compares ASOs with other RNA modalities including siRNA, miRNA modulators, mRNA therapeutics, aptamers, shRNA, and CRISPR/Cas-guided single-guide RNAs.

Source:

The review contrasts ASOs with RNAi, siRNAs, CRISPR, optogenetics, chemogenetics, and virus-based delivery systems.

Compared with CRISPR/Cas9 system

The review compares ASOs with other RNA modalities including siRNA, miRNA modulators, mRNA therapeutics, aptamers, shRNA, and CRISPR/Cas-guided single-guide RNAs.; The review contrasts ASOs with RNAi, siRNAs, CRISPR, optogenetics, chemogenetics, and virus-based delivery systems.

Shared frame: source-stated alternative in extracted literature

Strengths here: highlighted as a major RNA therapeutic modality; given special emphasis for neurological, metabolic, and infectious diseases; explicitly named as a therapeutic component class in the supplied summary.

Relative tradeoffs: broader clinical application is limited by translational barriers; common challenges include in vivo stability, delivery efficiency, and immune activation; the provided payload does not specify exact target classes, chemistries, or delivery dependencies from the anchor review.

Source:

The review compares ASOs with other RNA modalities including siRNA, miRNA modulators, mRNA therapeutics, aptamers, shRNA, and CRISPR/Cas-guided single-guide RNAs.

Source:

The review contrasts ASOs with RNAi, siRNAs, CRISPR, optogenetics, chemogenetics, and virus-based delivery systems.

Compared with Exosomes

The supplied summary points to exosomes, nanoparticles, liposomes, and aptamer-siRNA systems as adjacent approaches.

Shared frame: source-stated alternative in extracted literature

Strengths here: highlighted as a major RNA therapeutic modality; given special emphasis for neurological, metabolic, and infectious diseases; explicitly named as a therapeutic component class in the supplied summary.

Relative tradeoffs: broader clinical application is limited by translational barriers; common challenges include in vivo stability, delivery efficiency, and immune activation; the provided payload does not specify exact target classes, chemistries, or delivery dependencies from the anchor review.

Source:

The supplied summary points to exosomes, nanoparticles, liposomes, and aptamer-siRNA systems as adjacent approaches.

Compared with guide RNA

The review compares ASOs with other RNA modalities including siRNA, miRNA modulators, mRNA therapeutics, aptamers, shRNA, and CRISPR/Cas-guided single-guide RNAs.

Shared frame: source-stated alternative in extracted literature

Strengths here: highlighted as a major RNA therapeutic modality; given special emphasis for neurological, metabolic, and infectious diseases; explicitly named as a therapeutic component class in the supplied summary.

Relative tradeoffs: broader clinical application is limited by translational barriers; common challenges include in vivo stability, delivery efficiency, and immune activation; the provided payload does not specify exact target classes, chemistries, or delivery dependencies from the anchor review.

Source:

The review compares ASOs with other RNA modalities including siRNA, miRNA modulators, mRNA therapeutics, aptamers, shRNA, and CRISPR/Cas-guided single-guide RNAs.

Compared with microRNA

The review compares ASOs with other RNA modalities including siRNA, miRNA modulators, mRNA therapeutics, aptamers, shRNA, and CRISPR/Cas-guided single-guide RNAs.

Shared frame: source-stated alternative in extracted literature

Strengths here: highlighted as a major RNA therapeutic modality; given special emphasis for neurological, metabolic, and infectious diseases; explicitly named as a therapeutic component class in the supplied summary.

Relative tradeoffs: broader clinical application is limited by translational barriers; common challenges include in vivo stability, delivery efficiency, and immune activation; the provided payload does not specify exact target classes, chemistries, or delivery dependencies from the anchor review.

Source:

The review compares ASOs with other RNA modalities including siRNA, miRNA modulators, mRNA therapeutics, aptamers, shRNA, and CRISPR/Cas-guided single-guide RNAs.

The review contrasts ASOs with RNAi, siRNAs, CRISPR, optogenetics, chemogenetics, and virus-based delivery systems.

Shared frame: source-stated alternative in extracted literature

Strengths here: highlighted as a major RNA therapeutic modality; given special emphasis for neurological, metabolic, and infectious diseases; explicitly named as a therapeutic component class in the supplied summary.

Relative tradeoffs: broader clinical application is limited by translational barriers; common challenges include in vivo stability, delivery efficiency, and immune activation; the provided payload does not specify exact target classes, chemistries, or delivery dependencies from the anchor review.

Source:

The review contrasts ASOs with RNAi, siRNAs, CRISPR, optogenetics, chemogenetics, and virus-based delivery systems.

The review contrasts ASOs with RNAi, siRNAs, CRISPR, optogenetics, chemogenetics, and virus-based delivery systems.

Shared frame: source-stated alternative in extracted literature

Strengths here: highlighted as a major RNA therapeutic modality; given special emphasis for neurological, metabolic, and infectious diseases; explicitly named as a therapeutic component class in the supplied summary.

Relative tradeoffs: broader clinical application is limited by translational barriers; common challenges include in vivo stability, delivery efficiency, and immune activation; the provided payload does not specify exact target classes, chemistries, or delivery dependencies from the anchor review.

Source:

The review contrasts ASOs with RNAi, siRNAs, CRISPR, optogenetics, chemogenetics, and virus-based delivery systems.

Compared with sgRNA

The review compares ASOs with other RNA modalities including siRNA, miRNA modulators, mRNA therapeutics, aptamers, shRNA, and CRISPR/Cas-guided single-guide RNAs.

Shared frame: source-stated alternative in extracted literature

Strengths here: highlighted as a major RNA therapeutic modality; given special emphasis for neurological, metabolic, and infectious diseases; explicitly named as a therapeutic component class in the supplied summary.

Relative tradeoffs: broader clinical application is limited by translational barriers; common challenges include in vivo stability, delivery efficiency, and immune activation; the provided payload does not specify exact target classes, chemistries, or delivery dependencies from the anchor review.

Source:

The review compares ASOs with other RNA modalities including siRNA, miRNA modulators, mRNA therapeutics, aptamers, shRNA, and CRISPR/Cas-guided single-guide RNAs.

Compared with small interfering RNA

The review compares ASOs with other RNA modalities including siRNA, miRNA modulators, mRNA therapeutics, aptamers, shRNA, and CRISPR/Cas-guided single-guide RNAs.; The supplied summary points to exosomes, nanoparticles, liposomes, and aptamer-siRNA systems as adjacent approaches.; The review contrasts ASOs with RNAi, siRNAs, CRISPR, optogenetics, chemogenetics, and virus-based delivery systems.

Shared frame: source-stated alternative in extracted literature

Strengths here: highlighted as a major RNA therapeutic modality; given special emphasis for neurological, metabolic, and infectious diseases; explicitly named as a therapeutic component class in the supplied summary.

Relative tradeoffs: broader clinical application is limited by translational barriers; common challenges include in vivo stability, delivery efficiency, and immune activation; the provided payload does not specify exact target classes, chemistries, or delivery dependencies from the anchor review.

Source:

The review compares ASOs with other RNA modalities including siRNA, miRNA modulators, mRNA therapeutics, aptamers, shRNA, and CRISPR/Cas-guided single-guide RNAs.

Source:

The supplied summary points to exosomes, nanoparticles, liposomes, and aptamer-siRNA systems as adjacent approaches.

Source:

The review contrasts ASOs with RNAi, siRNAs, CRISPR, optogenetics, chemogenetics, and virus-based delivery systems.

The review compares ASOs with other RNA modalities including siRNA, miRNA modulators, mRNA therapeutics, aptamers, shRNA, and CRISPR/Cas-guided single-guide RNAs.

Shared frame: source-stated alternative in extracted literature

Strengths here: highlighted as a major RNA therapeutic modality; given special emphasis for neurological, metabolic, and infectious diseases; explicitly named as a therapeutic component class in the supplied summary.

Relative tradeoffs: broader clinical application is limited by translational barriers; common challenges include in vivo stability, delivery efficiency, and immune activation; the provided payload does not specify exact target classes, chemistries, or delivery dependencies from the anchor review.

Source:

The review compares ASOs with other RNA modalities including siRNA, miRNA modulators, mRNA therapeutics, aptamers, shRNA, and CRISPR/Cas-guided single-guide RNAs.

Ranked Citations

  1. 1.
    StructuralSource 1Cell Reports Medicine2024Claim 2Claim 3Claim 4

    Seeded from load plan for claim cl1. Extracted from this source document.

  2. 2.
    StructuralSource 2PMC2026Claim 1

    Extracted from this source document.