Answer
Onasemnogene abeparvovec uses a non-replicating recombinant AAV vector with an AAV9 capsid to deliver a stable, fully functional human SMN transgene.
Sources for this answer
- Zolgensma 2 x 10Exp13 vector genomes/mL solution for infusionNovartis Pharmaceuticals UK Ltd · emc · SmPC §5.1 Pharmacodynamic properties · Version 17Document updated
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