Low-dose MyoAAV 2A-mediated delivery of engineered micro-utrophin achieves pan- muscle tissue distribution with elevated muscle function in Duchenne muscular dystrophy - PubMed
4 hours ago
- #Gene Therapy
- #Micro-utrophin
- #Duchenne Muscular Dystrophy
- A low-dose MyoAAV 2A capsid delivering engineered micro-utrophin achieved widespread muscle expression and improved function in DMD mouse models.
- The strategy reduced muscle damage, inflammation, and fibrosis while avoiding cardiac or liver toxicity.
- It overcomes the efficacy-toxicity trade-off common in DMD gene therapies using high-dose AAV vectors.