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Gene and cell‐mediated therapies for muscular dystrophy
Author(s) -
Konieczny Patryk,
Swiderski Kristy,
Chamberlain Jeffrey S.
Publication year - 2013
Publication title -
muscle and nerve
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.025
H-Index - 145
eISSN - 1097-4598
pISSN - 0148-639X
DOI - 10.1002/mus.23738
Subject(s) - duchenne muscular dystrophy , medicine , muscular dystrophy , dystrophin , genetic enhancement , disease , clinical trial , bioinformatics , neuromuscular disease , muscle disease , muscle disorder , physical medicine and rehabilitation , physical therapy , gene , pathology , biology , genetics
Duchenne muscular dystrophy ( DMD ) is a devastating muscle disorder that affects 1 in 3,500 boys. Despite years of research and considerable progress in understanding the molecular mechanism of the disease and advancement of therapeutic approaches, there is no cure for DMD . The current treatment options are limited to physiotherapy and corticosteroids, and although they provide a substantial improvement in affected children, they only slow the course of the disorder. On a more optimistic note, more recent approaches either significantly alleviate or eliminate muscular dystrophy in murine and canine models of DMD and importantly, many of them are being tested in early phase human clinical trials. This review summarizes advancements that have been made in viral and nonviral gene therapy as well as stem cell therapy for DMD with a focus on the replacement and repair of the affected dystrophin gene. Muscle Nerve 47: 649–663, 2013