Advance and cogitation of gene therapy for Duchenne muscular dystrophy

Abstract

Duchenne muscular dystrophy (DMD) is the common hereditary muscular disease caused by the deficiency of cytoskeletal protein dystrophin on the sarcolemma. It is characterized by progessive muscle weakness and atrophy and dying of heart or respiratory failure. Currently the gene therapy strategies of DMD can be catagorized into two groups: restoring dystrophin expression and compensating for the lack of dystrophin. Therapies restoring dystrophin include nonsense mutation readthrough, exon skipping, adeno-associated virus (AAV) mediated micro-dystrophin therapy and gene editing. Here we summarize the latest advance of gene therapy for DMD, focusing on strategies that restore dystrophin, hoping to benefit the choosing of optimal gene therapy.

DOI: 10.3969/j.issn.1672-6731.2019.05.004