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rAAV9-microdystrophin
- Development stage
- Preclinical
- Lead developer
- Sarepta Therapeutics
- Modality
- Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Intravenous
01
Overview
rAAV9-microdystrophin is a gene therapy utilizing a recombinant adeno-associated virus serotype 9 (rAAV9) vector to deliver a functional microdystrophin gene to muscle cells. This therapy is designed to address the underlying genetic deficiency in Duchenne muscular dystrophy (DMD) by restoring dystrophin protein function in skeletal and cardiac muscle. Microdystrophin, a truncated but functional version of dystrophin, is expressed following transduction, aiming to stabilize muscle fibers and improve muscle strength and integrity. The therapy leverages systemic delivery, and immune response modulation may enhance efficacy[1][2][3][4].
- Other names
- rAAV9-microdystrophinrAAV-9-microdystrophinrAAV 9-microdystrophinrAAV9-μDysrAAV9-CK8e-c-μDys5
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