Answer
Eladocagene exuparvovec delivers human DDC gene cDNA using a recombinant AAV2 vector infused into the putamen, enabling expression of the AADC enzyme, which converts L-DOPA to dopamine in patients with AADC deficiency.
Sources for this answer
- Upstaza (Eladocagene exuparvovec) solution for infusionPTC Therapeutics Ltd · emc · SmPC §5.1 Pharmacodynamic properties · Version 7Document updated
Sources checked