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zildistrogene varoparvovec
- Development stage
- Unknown
- Lead developer
- Sarepta Therapeutics
- Modality
- Recombinant Proteins and Enzymes, Gene Therapies
- Administration
- Intravenous
01
Overview
Zildistrogene varoparvovec is a gene therapy that uses a recombinant, non-replicating adeno-associated virus type 2/9 (rAAV2/9) vector to deliver the human microdystrophin gene under the control of a muscle-specific promoter. It is designed for the treatment of Duchenne muscular dystrophy (DMD), aiming to restore dystrophin protein expression in muscle cells. The therapy introduces a functional microdystrophin gene into patients' muscle tissue, compensating for the genetic mutations that cause DMD[2][3][4].
02
Targets
DMD (Dystrophin)
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