Generation and characterization of an induced pluripotent stem cell line (ZSYYDNi001-A) from a patient with Duchenne muscular dystrophy carrying exon 51 deletion in the DMD gene.

Abstract

Duchenne muscular dystrophy (DMD) is a common hereditary neuromuscular disease characterized by progressive muscle wasting and weakness. DMD is caused by mutations in the DMD gene, resulting in the dysfunction of dystrophin. We generated an induced pluripotent stem cell (iPSC) from a patient with DMD carrying exon 51 deletion in the DMD gene. This iPSC line can serve as a model for studying the pathogenesis and therapeutics of DMD.