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Research progress of therapies of Duchenne muscular dystrophy |
YANG Yixian1 LI Changsheng1 WANG Beilei1 DANG Suying2 |
1.School of Medicine, Shanghai Jiao Tong University, Shanghai 200025, China;
2.Department of Biochemistry and Molecular Cell Biology, School of Medicine, Shanghai Jiao Tong University, Shanghai 200025, China |
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Abstract Duchenne muscular dystrophy (DMD) is a kind of X-linked recessive hereditary disease caused by mutations of the dystrophin gene. There is no effective therapy method at present. In recent years, with the increasing knowledge of the mechanisms and pathologic process of the disease, researchers have conducted studies of DMD therapy method and the effectiveness. These studies include developing pharmaceutical therapies for reducing inflammation, regulating calcium levels, promoting muscle growth and anti-fibrosis, developing auxiliary equipment to train or improve the life quality of patients, developing gene therapy strategies for DMD gene replacement, exon-skipping and gene editing, and investigating stem cell therapy. This paper summarizes the latest progress of each therapy for DMD.
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