Source 1primary paper2026MED
Toolkit/lentiviral vectors
lentiviral vectors
Also known as: lentiviral-based interventions, lentiviral vectors, lentiviruses, LVVs, recombinant lentiviral construct
Taxonomy: Mechanism Branch / Architecture. Workflows sit above the mechanism and technique branches rather than replacing them.
Summary
Lentiviral vectors (LVVs) are used as a viral gene therapeutic and were derived from human immunodeficiency virus subtype 1 (HIV-1). LVVs are used to deliver and induce the stable expression of transgenes through genome integration.
Usefulness & Problems
No literature-backed usefulness or problem-fit explainer has been materialized for this record yet.
Published Workflows
Objective: Engineer and preliminarily evaluate a FAP-targeted CAR design for fibrosis-selective cytotoxicity against cardiac myofibroblasts.
Why it works: The workflow uses engineered Jurkat cells as a preliminary screening model to test whether the FAP-targeted 4-1BB CAR can be expressed and can mediate target recognition and cytotoxicity before moving to primary T cell models.
selective recognition of FAP-expressing cardiac myofibroblastsapoptosis induction in target myofibroblastscell engineering using lentiviral vectorscell engineering using lipid nanoparticlespreliminary screening in Jurkat cells
Stages
- 1.Generation of FAP-CAR-engineered Jurkat screening model(library_build)
This stage creates the engineered cell model needed for downstream evaluation of CAR expression and function.
Selection: Generate engineered Jurkat cells carrying the FAP-targeted CAR using lentiviral vectors or lipid nanoparticles.
- 2.Preliminary in vitro functional screening(broad_screen)
This stage tests whether the engineered CAR design is functional in vitro before further investigation in primary T cell models.
Selection: Evaluate CAR expression, target recognition, and in vitro cytotoxic activity in the engineered Jurkat model.
- 3.Target-cell apoptosis and cytokine assessment(confirmatory_validation)
This stage confirms target-specific functional activity and checks a safety-relevant cytokine signal in the in vitro system.
Selection: Confirm selective recognition and apoptosis induction in FAP-expressing cardiac myofibroblasts while checking for excessive IL-6 secretion.
Steps
- 1.Engineer Jurkat cells with the FAP-targeted 4-1BB CAR using two delivery approachesengineered construct
Create a preliminary screening model for evaluating the FAP-targeted CAR design.
The engineered Jurkat model must be generated before CAR expression and functional activity can be tested.
- 2.Evaluate CAR expression, target recognition, and in vitro cytotoxic activityengineered screening model
Determine whether the engineered CAR is expressed and functionally engages target cells in vitro.
Expression and basic functional screening are used as the preliminary evaluation of the construct before stronger claims about therapeutic relevance.
- 3.Assess apoptosis in FAP-expressing cardiac myofibroblasts and monitor IL-6 secretionengineered effector cells
Confirm fibrosis-selective cytotoxicity and check for a safety-relevant cytokine signal.
After establishing expression and cytotoxic activity, the study tests whether target-cell killing is accompanied by apoptosis induction without excessive IL-6 secretion.
Taxonomy & Function
Primary hierarchy
Mechanism Branch
Architecture: A delivery strategy grouped with the mechanism branch because it determines how a system is instantiated and deployed in context.
Techniques
Structural CharacterizationTarget processes
editingrecombinationtranscriptionInput: Chemical
Validation
Cell-freeBacteriaMammalianMouseHumanTherapeuticIndep. Replication
Supporting Sources
Source 2primary paper2025MED
Source 3review2025MED
Source 4review2015Frontiers in Molecular Neuroscience
Source 5review2016Journal of Neurophysiology
Source 6primary paper2025MED
Source 7primary paper2025MED
Ranked Claims
An increasingly popular application of lentiviral vectors is the generation of CAR T cell therapies that enhance T cell antigen specificity and effector function in liquid cancers.
An increasingly popular application of LVV is in the generation of chimeric antigen receptor (CAR) T cell therapies, which change and enhance T cell antigen specificity and effector function in liquid cancers.
Lentiviral vectors are used in FDA-approved therapies for diseases including beta thalassemia and sickle cell anemia.
Several Food and Drug Administration (FDA)-approved LVV-derived therapies are used for treating diseases ranging from beta thalassemia to sickle cell anemia.
Current clinical 3rd generation LVV transduction systems use a 4-plasmid combination encoding critical structural and non-structural HIV-1 proteins while excluding HIV-1 major accessory genes.
Current clinical LVV delivery systems do not include HIV-1 major accessory genes; however, critical structural and non-structural HIV-1 proteins are encoded by the 4-plasmid combination that composes the 3rd generation LVV transduction systems.
plasmid count 4
Lentiviral vectors deliver transgenes and induce stable expression through genome integration.
LVVs are used to deliver and induce the stable expression of transgenes through genome integration.
Investigation of LVV integration has uncovered chimeric LVV-host transcripts and altered host transcript splicing patterns.
Investigation of LVV integration has uncovered the generation of chimeric LVV-host transcripts and altered host transcript splicing patterns.
LVVs integrate into host intronic and intergenic regions due to genomic accessibility, with no known bias toward specific target integration motifs.
LVVs integrate into host intronic and intergenic regions due to genomic accessibility, but there are no known biases toward specific target integration motifs.
LVV integrations driving oncogene expression could be a cause for malignancy development.
LVV integrations driving oncogene expression could be a cause for malignancy development.
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.
Gene delivery approaches may advance conventional antibody therapeutics against viral infections and other diseases through local persistence of the proteins.
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.
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.
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.
mRNA platforms, viral vectors, and engineered cell therapies have matured considerably due to years of clinical experience and growing regulatory confidence.
Suicide gene strategies were effective in mitigating safety risks in the reviewed HSC engineering context.
Innovations in gene-delivery platforms including lentiviral vectors, AAV vectors, and lipid nanoparticles are opening avenues to modulate the hepatic tumor niche.
innovations in gene-delivery platforms, from lentiviral and AAV vectors to lipid nanoparticles, ... are opening avenues to modulate the hepatic tumor niche
Clinical success of AAV- and lentiviral-based interventions and approval of CAR-T cell therapies highlight the potential of these technologies to transform delivery of antibody therapeutics.
Anterograde projection targeting and retrograde transport of viral vectors are two projection-targeting approaches for probing neural circuits.
We discuss two projection targeting approaches for probing neural circuits: anterograde projection targeting and retrograde transport of viral vectors.
Cell type-specific promoters and other nucleotide sequences can be used in viral vectors to target neuronal types at the transcriptional level.
cell type-specific promoters and other nucleotide sequences that can be used in viral vectors to target neuronal types at the transcriptional level
Targeting specific neuronal types with optogenetic and chemogenetic tools remains challenging in primates.
In primates, however, targeting specific types of neurons with these tools remains challenging.
Viral-vector targeting performance is discussed in terms of cell-type tropism and prospects for improved efficacy and selectivity through new variants.
their tropism for different cell types, and prospects for new variants with improved efficacy and selectivity
AAV and lentiviral vectors are major viral-vector platforms discussed for gene delivery to neurons in the adult primate central nervous system.
We review the literature on viral vectors for gene delivery to neurons, focusing on adeno-associated viral vectors and lentiviral vectors
Combining lentiviral tract tracing with immunohistochemistry and confocal or electron microscopy enables selective labeling and tracing of specific neuronal populations at gross or ultrastructural levels.
Lentiviral vectors mediate efficient gene transfer to non-dividing cells, including neurons and glia in the adult mammalian brain.
New generation optogenetic technologies can be readily utilized with lentiviral vector approaches to analyze neuronal circuit and gene functions in the mature mammalian brain.
Integration of the recombinant lentiviral construct into the host genome provides permanent expression, including in progeny of dividing neural precursors.
Targeted lentiviral vectors can be built using modified envelope glycoproteins and cell-selective or inducible promoters.
Approval Evidence
7 sources20 linked approval claimsfirst-pass slug lentiviral-vectors
Lentiviral vectors (LVVs) are used as a viral gene therapeutic and were derived from human immunodeficiency virus subtype 1 (HIV-1). LVVs are used to deliver and induce the stable expression of transgenes through genome integration.
Source:
Advances in gene-editing technologies, such as CRISPR/Cas9 and lentiviral vectors, have enabled the modification of HSCs... Lentiviral vectors were the most common delivery method, but concerns about insertional mutagenesis persist.
Source:
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.
Source:
innovations in gene-delivery platforms, from lentiviral and AAV vectors to lipid nanoparticles
Source:
The clinical success of adeno-associated virus (AAV)- and lentiviral-based interventions ... highlight the potential of these technologies to transform how we deliver Ab therapeutics.
Source:
We review the literature on viral vectors for gene delivery to neurons, focusing on adeno-associated viral vectors and lentiviral vectors, their tropism for different cell types, and prospects for new variants with improved efficacy and selectivity.
Source:
Lentiviruses have been extensively used as gene delivery vectors since the mid-1990s. Usually derived from the human immunodeficiency virus genome, they mediate efficient gene transfer to non-dividing cells, including neurons and glia in the adult mammalian brain. In addition, integration of the recombinant lentiviral construct into the host genome provides permanent expression, including the progeny of dividing neural precursors.
Source:
applicationsupports
An increasingly popular application of lentiviral vectors is the generation of CAR T cell therapies that enhance T cell antigen specificity and effector function in liquid cancers.
An increasingly popular application of LVV is in the generation of chimeric antigen receptor (CAR) T cell therapies, which change and enhance T cell antigen specificity and effector function in liquid cancers.
Source:
applicationsupports
Lentiviral vectors are used in FDA-approved therapies for diseases including beta thalassemia and sickle cell anemia.
Several Food and Drug Administration (FDA)-approved LVV-derived therapies are used for treating diseases ranging from beta thalassemia to sickle cell anemia.
Source:
functionsupports
Lentiviral vectors deliver transgenes and induce stable expression through genome integration.
LVVs are used to deliver and induce the stable expression of transgenes through genome integration.
Source:
host impactsupports
Investigation of LVV integration has uncovered chimeric LVV-host transcripts and altered host transcript splicing patterns.
Investigation of LVV integration has uncovered the generation of chimeric LVV-host transcripts and altered host transcript splicing patterns.
Source:
integration patternsupports
LVVs integrate into host intronic and intergenic regions due to genomic accessibility, with no known bias toward specific target integration motifs.
LVVs integrate into host intronic and intergenic regions due to genomic accessibility, but there are no known biases toward specific target integration motifs.
Source:
safety risksupports
LVV integrations driving oncogene expression could be a cause for malignancy development.
LVV integrations driving oncogene expression could be a cause for malignancy development.
Source:
application demosupports
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.
Source:
application scopesupports
Gene delivery approaches may advance conventional antibody therapeutics against viral infections and other diseases through local persistence of the proteins.
Source:
delivery tradeoffmixed
Lentiviral vectors were the most common delivery method in the reviewed studies, but insertional mutagenesis remains a concern.
Source:
maturity statementsupports
mRNA platforms, viral vectors, and engineered cell therapies have matured considerably due to years of clinical experience and growing regulatory confidence.
Source:
therapeutic opportunitysupports
Innovations in gene-delivery platforms including lentiviral vectors, AAV vectors, and lipid nanoparticles are opening avenues to modulate the hepatic tumor niche.
innovations in gene-delivery platforms, from lentiviral and AAV vectors to lipid nanoparticles, ... are opening avenues to modulate the hepatic tumor niche
Source:
translation potentialsupports
Clinical success of AAV- and lentiviral-based interventions and approval of CAR-T cell therapies highlight the potential of these technologies to transform delivery of antibody therapeutics.
Source:
challenge statementsupports
Targeting specific neuronal types with optogenetic and chemogenetic tools remains challenging in primates.
In primates, however, targeting specific types of neurons with these tools remains challenging.
Source:
property summarysupports
Viral-vector targeting performance is discussed in terms of cell-type tropism and prospects for improved efficacy and selectivity through new variants.
their tropism for different cell types, and prospects for new variants with improved efficacy and selectivity
Source:
scope summarysupports
AAV and lentiviral vectors are major viral-vector platforms discussed for gene delivery to neurons in the adult primate central nervous system.
We review the literature on viral vectors for gene delivery to neurons, focusing on adeno-associated viral vectors and lentiviral vectors
Source:
application summarysupports
Combining lentiviral tract tracing with immunohistochemistry and confocal or electron microscopy enables selective labeling and tracing of specific neuronal populations at gross or ultrastructural levels.
Source:
capability summarysupports
Lentiviral vectors mediate efficient gene transfer to non-dividing cells, including neurons and glia in the adult mammalian brain.
Source:
compatibility summarysupports
New generation optogenetic technologies can be readily utilized with lentiviral vector approaches to analyze neuronal circuit and gene functions in the mature mammalian brain.
Source:
durability summarysupports
Integration of the recombinant lentiviral construct into the host genome provides permanent expression, including in progeny of dividing neural precursors.
Source:
targeting strategy summarysupports
Targeted lentiviral vectors can be built using modified envelope glycoproteins and cell-selective or inducible promoters.
Source:
Comparisons
No literature-backed comparison notes have been materialized for this record yet.
Ranked Citations