Update on the Treatment of Duchenne Muscular Dystrophy

Nerve and Muscle (M Hirano and LH Weimer, Section Editors)

DOI: 10.1007/s11910-012-0332-1

Cite this article as:
Rodino-Klapac, L.R., Mendell, J.R. & Sahenk, Z. Curr Neurol Neurosci Rep (2013) 13: 332. doi:10.1007/s11910-012-0332-1
Part of the following topical collections:
  1. Topical Collection on Nerve and Muscle

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.

Keywords

Duchenne muscular dystrophy Gene therapy Exon skipping Stem cell therapy Newborn screening 

Copyright information

© Springer Science+Business Media New York 2013

Authors and Affiliations

  1. 1.Department of PediatricsThe Ohio State University, and Nationwide Children’s HospitalColumbusUSA
  2. 2.Department of NeurologyThe Ohio State University, and Nationwide Children’s HospitalColumbusUSA
  3. 3.Center for Gene Therapy at The Research Institute at Nationwide Children’s HospitalColumbusUSA

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