Gene therapeutic strategies and relevant clinical trials in neuromuscular disorder in China.

Neuromuscular disorder is a diverse group of genetic disease, among which Duchenne muscular dystrophy and Spinal muscular atrophy are most common. Recently, the great breakthroughs of gene targeted therapeutic strategies are leading a profound revolution in the standard care of neuromuscular disorders over the world including China. This review will offer an outline of the molecular pathogenesis, clinical progress, critical trials, as well as the challenges of new gene therapy in the treatment of Spinal muscular atrophy and Duchenne muscular dystrophy in China, mainly includes mRNA splicing modulators and adeno-associated virus mediated gene replacement. We hope to highlight some important findings about the critical development of gene therapy in this field, which might be helpful for suggesting potential therapeutic treatment for neuromuscular disease in China.