ArticleMolecular therapy : the journal of the American Society of Gene Therapy2012
Directed evolution of adeno-associated virus for enhanced gene delivery and gene targeting in human pluripotent stem cells.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 74 papers.
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The trial behind it
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Who cites it
74 citing papers in PubMed, 119 citations in OpenAlex.
- AAV Vectors in Regenerative Medicine and Cellular Reprogramming: Potential, Pitfalls, and Specificity Constraints.International journal of molecular sciences · 2026Review
- Navigating the Hurdles of Intra-Articular AAV Gene Therapy.International journal of molecular sciences · 2025Review
- Adeno-Associated Virus-Engineered Umbilical Cord-Derived Mesenchymal Stromal Cells Overexpressing Human sFlt-1 for Anti-Angiogenesis.Life (Basel, Switzerland) · 2025Article
- Design and Characterization of a Novel Intravitreal Dual-Transgene Genetic Medicine for Neovascular Retinopathies.Investigative ophthalmology & visual science · 2024Article
- Designing molecules: directing stem cell differentiation.Frontiers in bioengineering and biotechnology · 2024Review
- Identification and validation of novel engineered AAV capsid variants targeting human glia.Frontiers in neuroscience · 2024Article
- Optical Penetration of Shape-Controlled Metallic Nanosensors across Membrane Barriers.Sensors (Basel, Switzerland) · 2023Article
- Mitigating Serious Adverse Events in Gene Therapy with AAV Vectors: Vector Dose and Immunosuppression.Drugs · 2023Review
- PEG-mediated transduction of rAAV as a platform for spatially confined and efficient gene delivery.Biomaterials research · 2022Article
- Identification of adeno-associated virus variants for gene transfer into human neural cell types by parallel capsid screening.Scientific reports · 2022Article
- Vector engineering, strategies and targets in cancer gene therapy.Cancer gene therapy · 2022Review
- Intracellular virion traffic to the endosome driven by cell type specific sialic acid receptors determines parvovirus tropism.Frontiers in microbiology · 2022Article
- Advances in designing Adeno-associated viral vectors for development of anti-HBV gene therapeutics.Virology journal · 2021Review
- Synthetic Biology: Emerging Concepts to Design and Advance Adeno-Associated Viral Vectors for Gene Therapy.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2021Review
- Applying machine learning to predict viral assembly for adeno-associated virus capsid libraries.Molecular therapy. Methods & clinical development · 2021Article
- The Potential of Induced Pluripotent Stem Cells to Test Gene Therapy Approaches for Neuromuscular and Motor Neuron Disorders.Frontiers in cell and developmental biology · 2021Review
- CIRM tools and technologies: Breaking bottlenecks to the development of stem cell therapies.Stem cells translational medicine · 2020Article
- Adeno-Associated Virus-Based Gene Therapy for Lifelong Correction of Genetic Disease.Human gene therapy · 2020Review
- Anterograde Neuronal Circuit Tracers Derived from Herpes Simplex Virus 1: Development, Application, and Perspectives.International journal of molecular sciences · 2020Review
- Translating CRISPR-Cas Therapeutics: Approaches and Challenges.The CRISPR journal · 2020Review
14 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors at 1 institution in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Efficient approaches for the precise genetic engineering of human pluripotent stem cells (hPSCs) can enhance both basic and applied stem cell research. Adeno- associated virus (AAV) vectors are of particular interest for their capacity to mediate efficient gene delivery to and gene targeting in various cells. However, natural AAV serotypes offer only modest transduction of human embryonic and induced pluripotent stem cells (hESCs and hiPSCs), which limits their utility for efficiently manipulating the hPSC genome. Directed evolution is a powerful means to generate viral vectors with novel capabilities, and we have applied this approach to create a novel AAV variant with high gene delivery efficiencies (~50%) to hPSCs, which are importantly accompanied by a considerable increase in gene-targeting frequencies, up to 0.12%. While this level is likely sufficient for numerous applications, we also show that the gene-targeting efficiency mediated by an evolved AAV variant can be further enhanced (>1%) in the presence of targeted double- stranded breaks (DSBs) generated by the co-delivery of artificial zinc finger nucleases (ZFNs). Thus, this study demonstrates that under appropriate selective pressures, AAV vectors can be created to mediate efficient gene targeting in hPSCs, alone or in the presence of ZFN- mediated double-stranded DNA breaks.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.