Promising Treatments for Duchenne Muscular Dystrophy: Restoring Dystrophin Protein Expression Using Nucleic Acid Therapeutics

Abstract

Review Promising Treatments for Duchenne Muscular Dystrophy: Restoring Dystrophin Protein Expression Using Nucleic Acid Therapeutics Guo Hu  and Chen Chen * Division of Cardiology and Hubei Key Laboratory of Genetics and Molecular Mechanisms of Cardiological Disorders, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China. * Correspondence: chenchen@tjh.tjmu.edu.cn; Tel. & Fax: 86-27-6937-8422     Received: 10 October 2022 Accepted: 4 November 2022 Published: 11 January 2023   Abstract: Duchenne muscular dystrophy is caused by inadequate generation of functional dystrophin protein. Traditional clinical treatments can only slightly mitigate the progression of the disease, but not completely stem or reverse the decline in muscle function. Therapies aimed at dystrophin recovery are currently under development, among which are exon skipping and stop codon readthrough therapies. They are now used in clinics, while gene addition therapies are in phase III clinical trials. Gene editing therapies have also been approved for the first clinical trial recently. This review will discuss these emerging therapies, clinical trials, and directions for future developments.