Research progress of adeno-associated virus vector-mediated micro-dystrophin gene therapy for Duchenne muscular dystrophy

Abstract

Duchenne muscular dystrophy (DMD) is one of the most common single gene hereditary disease caused by mutation of DMD gene. How to replace the mutated gene has been greatly concerned until now. The continuous progress of gene therapy on DMD has focusing on constructing the micro-dystrophin and choosing a vector to transfer it through the whole body. Adeno-associated virus (AAV) has been widely used in this study because of its less pathogenicity, low immunogenicity and long-term expression in nondividing cells. This paper discussed the research progress of AAV micro-dystrophin gene therapy in DMD from the aspects of construction of AAV micro-dystrophin, animal models and clinical trials. In a conclusion, there is a long way to explore the clinical use of AAV micro?dystrophin on DMD patients, but it probably would be the most potential one we should pay more attention to.

DOI: 10.3969/j.issn.1672-6731.2019.05.005