Systemic Delivery of an AAV9 Exon-Skipping Vector Significantly Improves or Prevents Features of Duchenne Muscular Dystrophy in the Dup2 Mouse.
Molecular Therapy — Methods & Clinical Development（2022）
Key words
Duchenne muscular dystrophy,Becker muscular dystrophy,dystrophin,exon skipping,U7snRNA,gene therapy
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