ArticleMolecular therapy : the journal of the American Society of Gene Therapy2011
An evolved adeno-associated viral variant enhances gene delivery and gene targeting in neural stem cells.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 47 papers.
What it found
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Who cites it
47 citing papers in PubMed, 100 citations in OpenAlex.
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- Design and Characterization of a Novel Intravitreal Dual-Transgene Genetic Medicine for Neovascular Retinopathies.Investigative ophthalmology & visual science · 2024Article
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- Optimal trade-off control in machine learning-based library design, with application to adeno-associated virus (AAV) for gene therapy.Science advances · 2024Article
- Designing molecules: directing stem cell differentiation.Frontiers in bioengineering and biotechnology · 2024Review
- rAAV2-Mediated Restoration of GALC in Neural Stem Cells from Krabbe Patient-Derived iPSCs.Pharmaceuticals (Basel, Switzerland) · 2023Article
- Review
- Efficient spatially targeted gene editing using a near-infrared activatable protein-conjugated nanoparticle for brain applications.Nature communications · 2022Article
- Identification of adeno-associated virus variants for gene transfer into human neural cell types by parallel capsid screening.Scientific reports · 2022Article
- Widespread labeling and genomic editing of the fetal central nervous system byDevelopment (Cambridge, England) · 2021Article
- Emerging Concepts in Vector Development for Glial Gene Therapy: Implications for Leukodystrophies.Frontiers in cellular neuroscience · 2021Review
- 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
- Adeno-Associated Virus-Based Gene Therapy for Lifelong Correction of Genetic Disease.Human gene therapy · 2020Review
- AAV Capsid-Promoter Interactions Determine CNS Cell-Selective Gene Expression In Vivo.Molecular therapy : the journal of the American Society of Gene Therapy · 2020Article
- Viral Vectors, Animal Models, and Cellular Targets for Gene Therapy of Cystic Fibrosis Lung Disease.Human gene therapy · 2020Review
- AAVR-Displaying Interfaces: Serotype-Independent Adeno-Associated Virus Capture and Local Delivery Systems.Molecular therapy. Nucleic acids · 2019Article
- Safety and efficacy evaluations of an adeno-associated virus variant for preparing IL10-secreting human neural stem cell-based therapeutics.Gene therapy · 2019Article
- Capsid Modifications for Targeting and Improving the Efficacy of AAV Vectors.Molecular therapy. Methods & clinical development · 2019Review
- Engineered viral vectors for functional interrogation, deconvolution, and manipulation of neural circuits.Current opinion in neurobiology · 2018Review
- Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity.Molecular therapy : the journal of the American Society of Gene Therapy · 2018Article
Corrections and comments
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Authors and funding
10 authors at 3 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Gene delivery to, and gene targeting in, stem cells would be a highly enabling technology for basic science and biomedical application. Adeno-associated viral (AAV) vectors have demonstrated the capacity for efficient delivery to numerous cells, but their application to stem cells has been limited by low transduction efficiency. Due to their considerable advantages, however, engineering AAV delivery systems to enhance gene delivery to stem cells may have an impact in stem cell biology and therapy. Therefore, using several diverse AAV capsid libraries-including randomly mutagenized, DNA shuffled, and random peptide insertion variants-we applied directed evolution to create a "designer" AAV vector with enhanced delivery efficiency for neural stem cells (NSCs). A novel AAV variant, carrying an insertion of a selected peptide sequence on the surface of the threefold spike within the heparin-binding site, emerged from this evolution. Importantly, this evolved AAV variant mediated efficient gene delivery to rat, mouse, and human NSCs, as well as efficient gene targeting within adult NSCs, and it is thus promising for applications ranging from basic stem cell biology to clinical translation.
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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.