ArticleMolecular therapy. Methods & clinical development2018
Bioengineered Viral Platform for Intramuscular Passive Vaccine Delivery to Human Skeletal Muscle.
Article in Molecular therapy. Methods & clinical development, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.
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Who cites it
13 citing papers in PubMed.
- Recent Advancements in AAV-Vectored Immunoprophylaxis in the Nonhuman Primate Model.Biomedicines · 2023Review
- A strategy for high antibody expression with low anti-drug antibodies using AAV9 vectors.Frontiers in immunology · 2023Article
- Selection of rAAV vectors that cross the human blood-brain barrier and target the central nervous system using a transwell model.Molecular therapy. Methods & clinical development · 2022Article
- Ancestral library identifies conserved reprogrammable liver motif on AAV capsid.Cell reports. Medicine · 2022Article
- Identification of Broadly Applicable Adeno-Associated Virus Vectors by Systematic Comparison of Commonly Used Capsid VariantsHuman gene therapy · 2022Article
- Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders.Science advances · 2022Article
- Engineered bacterial voltage-gated sodium channel platform for cardiac gene therapy.Nature communications · 2022Article
- AAV vectors engineered to target insulin receptor greatly enhance intramuscular gene delivery.Molecular therapy. Methods & clinical development · 2020Article
- Methods Matter: Standard Production Platforms for Recombinant AAV Produce Chemically and Functionally Distinct Vectors.Molecular therapy. Methods & clinical development · 2020Article
- Multiplexed Cre-dependent selection yields systemic AAVs for targeting distinct brain cell types.Nature methods · 2020Article
- High-ThroughputHuman gene therapy · 2020Article
- Selection of an Efficient AAV Vector for Robust CNS Transgene Expression.Molecular therapy. Methods & clinical development · 2019Article
- Using a barcoded AAV capsid library to select for clinically relevant gene therapy vectors.JCI insight · 2019Article
Corrections and comments
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Authors and funding
14 authors.
Funding
Abstract
Skeletal muscle is ideal for passive vaccine administration as it is easily accessible by intramuscular injection. Recombinant adeno-associated virus (rAAV) vectors are in consideration for passive vaccination clinical trials for HIV and influenza. However, greater human skeletal muscle transduction is needed for therapeutic efficacy than is possible with existing serotypes. To bioengineer capsids with therapeutic levels of transduction, we utilized a directed evolution approach to screen libraries of shuffled AAV capsids in pools of surgically resected human skeletal muscle cells from five patients. Six rounds of evolution were performed in various muscle cell types, and evolved variants were validated against existing muscle-tropic serotypes rAAV1, 6, and 8. We found that evolved variants NP22 and NP66 had significantly increased primary human and rhesus skeletal muscle fiber transduction from surgical explants
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