Bioengineered AAV9 and Optimised Microdystrophin Vectors Augment Phenotypic Rescue in a Murine Model of Duchenne Muscular Dystrophy - PubMed
5 days ago
- #gene therapy
- #Duchenne muscular dystrophy
- #AAV9
- Bioengineered AAV9 and optimized microdystrophin vectors enhance phenotypic rescue in a Duchenne muscular dystrophy (DMD) murine model.
- The study found that a combination of AAV9 capsid engineering, optimal promoter selection (CAG promoter), and codon-optimization of microdystrophin improved dystrophin expression and muscle function.
- Intramuscular administration of engineered AAV9 vectors (N57Q, K51Q) with CAG-μDys significantly improved grip strength in mdx mice up to 18 weeks post-treatment.
- Systemic administration of AAV9K51Q vectors showed long-term benefits, including increased muscle contraction force (1.6-1.7 fold) and 30%-60% dystrophin restoration in skeletal and cardiac muscles up to 14 months post-treatment.
- The study highlights the therapeutic potential of engineered AAV9 vectors for clinical application in DMD patients.