CRISPR hfCas12Max mediated gene therapy restores dystrophin expression and muscle performance in DMD mice to support the initiation of MUSCLE clinical trial
Duchenne muscular dystrophy (DMD), an X-linked disease arising from genetic mutations in the dystrophin gene, has become the most common neuromuscular disorder in childhood. However, there is no curative therapy for this devastating disease at present. It is a huge unmet medical need to explore innovative yet efficient therapeutic approaches to correct genetic mutations and improve motor functions. Here, we generated a novel humanized DMD mice harboring deletion of mouse exon51 and exon52 via replacement with human exon51, which disrupted the dystrophin open reading frames.