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Molecular, cellular, and pharmacological therapies for Duchenne/Becker muscular dystrophies.

Publication ,  Journal Article
Chakkalakal, JV; Thompson, J; Parks, RJ; Jasmin, BJ
Published in: FASEB J
June 2005

Although the molecular defect causing Duchenne/Becker muscular dystrophy (DMD/BMD) was identified nearly 20 years ago, the development of effective therapeutic strategies has nonetheless remained a daunting challenge. Over the years, a variety of different approaches have been explored in an effort to compensate for the lack of the DMD gene product called dystrophin. This review not only presents some of the most promising molecular, cellular, and pharmacological strategies but also highlights some issues that need to be addressed before considering their implementation. Specifically, we describe current strategies being developed to exogenously deliver healthy copies of the dystrophin gene to dystrophic muscles. We present the findings of several studies that have focused on repairing the mutant dystrophin gene using various approaches. We include a discussion of cell-based therapies that capitalize on the use of myoblast or stem cell transfer. Finally, we summarize the results of several studies that may eventually lead to the development of appropriate drug-based therapies. In this context, we review our current knowledge of the mechanisms regulating expression of utrophin, the autosomal homologue of dystrophin. Given the complexity associated with the dystrophic phenotype, it appears likely that a combinatorial approach involving different therapeutic strategies will be necessary for the appropriate management and eventual treatment of this devastating neuromuscular disease.

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Published In

FASEB J

DOI

EISSN

1530-6860

Publication Date

June 2005

Volume

19

Issue

8

Start / End Page

880 / 891

Location

United States

Related Subject Headings

  • Utrophin
  • Transforming Growth Factor beta
  • Stem Cell Transplantation
  • Myostatin
  • Myoblasts
  • Mutation
  • Muscular Dystrophy, Duchenne
  • Humans
  • Glucocorticoids
  • Genetic Vectors
 

Citation

APA
Chicago
ICMJE
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Chakkalakal, J. V., Thompson, J., Parks, R. J., & Jasmin, B. J. (2005). Molecular, cellular, and pharmacological therapies for Duchenne/Becker muscular dystrophies. FASEB J, 19(8), 880–891. https://doi.org/10.1096/fj.04-1956rev
Chakkalakal, Joe V., Jennifer Thompson, Robin J. Parks, and Bernard J. Jasmin. “Molecular, cellular, and pharmacological therapies for Duchenne/Becker muscular dystrophies.FASEB J 19, no. 8 (June 2005): 880–91. https://doi.org/10.1096/fj.04-1956rev.
Chakkalakal JV, Thompson J, Parks RJ, Jasmin BJ. Molecular, cellular, and pharmacological therapies for Duchenne/Becker muscular dystrophies. FASEB J. 2005 Jun;19(8):880–91.
Chakkalakal, Joe V., et al. “Molecular, cellular, and pharmacological therapies for Duchenne/Becker muscular dystrophies.FASEB J, vol. 19, no. 8, June 2005, pp. 880–91. Pubmed, doi:10.1096/fj.04-1956rev.
Chakkalakal JV, Thompson J, Parks RJ, Jasmin BJ. Molecular, cellular, and pharmacological therapies for Duchenne/Becker muscular dystrophies. FASEB J. 2005 Jun;19(8):880–891.

Published In

FASEB J

DOI

EISSN

1530-6860

Publication Date

June 2005

Volume

19

Issue

8

Start / End Page

880 / 891

Location

United States

Related Subject Headings

  • Utrophin
  • Transforming Growth Factor beta
  • Stem Cell Transplantation
  • Myostatin
  • Myoblasts
  • Mutation
  • Muscular Dystrophy, Duchenne
  • Humans
  • Glucocorticoids
  • Genetic Vectors