Systemic delivery of rAAVrh74.MHCK7.micro-dystrophin in children with Duchenne muscular dystrophy

Takeaway

  • Single-dose AAVrh74.MHCK7.micro-dystrophin in Duchenne muscular dystrophy (DMD) patients has a favorable safety profile and provides clinical and biological benefits.

Why this matters

  • Gene transfer therapies show marked efficacy, tolerability and safety in children with neuromuscular diseases; however, this approach has not yet been tested in DMD due to the large length of the dystrophin gene.

  • Recently, researchers developed a micro-dystrophin transgene which could have an impact on patient symptomatology and life expectancy; clinical trials of this therapy are needed.