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Current Neurology and Neuroscience Reports
Published in: Current Neurology and Neuroscience Reports 3/2013

01-03-2013 | Nerve and Muscle (M Hirano and LH Weimer, Section Editors)

Update on the Treatment of Duchenne Muscular Dystrophy

Authors: Louise R. Rodino-Klapac, Jerry R. Mendell, Zarife Sahenk

Published in: Current Neurology and Neuroscience Reports | Issue 3/2013

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Abstract

Duchenne muscular dystrophy is the most severe childhood form of muscular dystrophy caused by mutations in the gene responsible for dystrophin production. There is no cure, and treatment is limited to glucocorticoids that prolong ambulation and drugs to treat the cardiomyopathy. Multiple treatment strategies are under investigation and have shown promise for Duchenne muscular dystrophy. Use of molecular-based therapies that replace or correct the missing or nonfunctional dystrophin protein has gained momentum. These strategies include gene replacement with adeno-associated virus, exon skipping with antisense oligonucleotides, and mutation suppression with compounds that “read through” stop codon mutations. Other strategies include cell therapy and surrogate gene products to compensate for the loss of dystrophin. All of these approaches are discussed in this review, with particular emphasis on the most recent advances made in each therapeutic discipline. The advantages of each approach and challenges in translation are outlined in detail. Individually or in combination, all of these therapeutic strategies hold great promise for treatment of this devastating childhood disease.
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Metadata
Title
Update on the Treatment of Duchenne Muscular Dystrophy
Authors
Louise R. Rodino-Klapac
Jerry R. Mendell
Zarife Sahenk
Publication date
01-03-2013
Publisher
Current Science Inc.
Published in
Current Neurology and Neuroscience Reports / Issue 3/2013
Print ISSN: 1528-4042
Electronic ISSN: 1534-6293
DOI
https://doi.org/10.1007/s11910-012-0332-1

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