Adeno-Associated Virus (AAV) Gene Delivery: Dissecting Molecular Interactions upon Cell Entry
Open Access
Abstract
Human gene therapy has advanced from twentieth-century conception to twenty-first-century reality. The recombinant Adeno-Associated Virus (rAAV) is a major gene therapy vector. Research continues to improve rAAV safety and efficacy using a variety of AAV capsid modification strategies. Significant factors influencing rAAV transduction efficiency include neutralizing antibodies, attachment factor interactions and receptor binding. Advances in understanding the molecular interactions during rAAV cell entry combined with improved capsid modulation strategies will help guide the design and engineering of safer and more efficient rAAV gene therapy vectors.Keywords
Funding Information
- National Institutes of Health (R35 GM1222564)
This publication has 199 references indexed in Scilit:
- Identification of the heparin binding site on adeno-associated virus serotype 3B (AAV-3B)Virology, 2012
- Adeno-Associated Virus 2 Infection Requires Endocytosis through the CLIC/GEEC PathwayCell Host & Microbe, 2011
- Systemic Gene Delivery in Large Species for Targeting Spinal Cord, Brain, and Peripheral Tissues for Pediatric DisordersMolecular Therapy, 2011
- Structure–function analysis of receptor-binding in adeno-associated virus serotype 6 (AAV-6)Virology, 2011
- Phospholipase A2Enzymes: Physical Structure, Biological Function, Disease Implication, Chemical Inhibition, and Therapeutic InterventionChemical Reviews, 2011
- The structure of adeno-associated virus serotype 3B (AAV-3B): Insights into receptor binding and immune evasionVirology, 2010
- Intravenous Administration of Self-complementary AAV9 Enables Transgene Delivery to Adult Motor NeuronsMolecular Therapy, 2009
- Adeno-associated virus-2 and its primary cellular receptor—Cryo-EM structure of a heparin complexVirology, 2009
- DNA Shuffling of Adeno-associated Virus Yields Functionally Diverse Viral ProgenyMolecular Therapy, 2008
- Making antibody fragments using phage display librariesNature, 1991