AAV2 Serotype
First AAV serotype discovered and most extensively characterized. Gold standard for AAV research with broad tissue tropism. Foundation for most engineered AAV variants.
Characteristics
Most studied AAV serotype. Uses HSPG as primary receptor. Broad tissue tropism but modest transduction efficiency. High pre-existing immunity (~60-80% seropositive in humans). Efficient transduction of neurons, photoreceptors, hepatocytes. Slow onset of transgene expression (2-4 weeks).
Applications
Retinal gene therapy (Luxturna - FDA approved for RPE65-associated retinal dystrophy). Hemophilia B (early trials). Parkinson's disease (AADC deficiency). CNS research tool. Proof-of-concept studies. Foundation for capsid engineering.
Literature References
- Russell et al. (2017). Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy. Lancet - Russell 2017 Luxturna
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