AAV9 Serotype
Breakthrough serotype capable of crossing the blood-brain barrier systemically. Revolutionized CNS gene therapy with broad neuronal and glial transduction. FDA-approved for spinal muscular atrophy.
Characteristics
Crosses blood-brain barrier after systemic administration. Broad CNS transduction (neurons, astrocytes, motor neurons). Efficient cardiac and skeletal muscle transduction. Uses terminal galactose as receptor. Age-dependent BBB penetration (more efficient in neonates). Moderate pre-existing immunity. Foundation for PHP and myoAAV engineering.
Applications
Spinal muscular atrophy (Zolgensma - FDA approved, most expensive drug). Giant axonal neuropathy (clinical trials). Duchenne muscular dystrophy. CLN6 Batten disease. Cardiac gene therapy. Most successful CNS gene therapy vector to date.
Literature References
- Mendell et al. (2017). Single-dose gene-replacement therapy for spinal muscular atrophy. N Engl J Med - Mendell 2017 Zolgensma
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