Advances in the treatment of Duchenne muscular dystrophy / 国际儿科学杂志

ABSTRACT

Duchenne muscular dystrophy(DMD) is the most common fx-linked recessive inherited myopathy,which is characterized by progressive aggravation,debilitating and lethal prognosis.There is no effective curative treatment for DMD.Classical corticosteroids therapy combined with physical training and multi-disciplinary comprehensive healthcare can effectively slow the disease progression and prolong life expectancy effectively.However,it cant change the outcome of patients.New alternative cell therapy,such as bone marrow mesenchymal stem cell transplantation has a certain effect,but there are many complications and the efficacy is uncertain.Skeletal muscle satellite cell transplantation is facing the problem of cell survival and migration.Gene therapy is the most promising treatment for DMD.Traditional methods include viral-mediated mini-or micro-DMD gene replacement,exon skipping strategies.CRISPR/Cas9-based genome editing technique is a promising treatment to cure the disease.Additionally,some new treatment targets of DMD such as gene Jaggedl and P2RX7,as well as small molecular therapies such as ARM210 and SMT C1100 have also been studied.In this review,recent progress in the treatment of DMD was summarized.