Journal of Molecular Medicine

, Volume 82, Issue 2, pp 102–115

Therapeutics for Duchenne muscular dystrophy: current approaches and future directions

  • Sasha Bogdanovich
  • Kelly J. Perkins
  • Thomas O. B. Krag
  • Tejvir S. Khurana
Review

DOI: 10.1007/s00109-003-0484-1

Cite this article as:
Bogdanovich, S., Perkins, K.J., Krag, T.O.B. et al. J Mol Med (2004) 82: 102. doi:10.1007/s00109-003-0484-1

Abstract

Duchenne muscular dystrophy (DMD) is the most common X-linked neuromuscular disorder. The devastating nature of DMD has led to an intense effort toward finding a cure for this disease, dating back to the time when Duchenne first initiated clinical trials using faradic stimulation for DMD patients. Unfortunately despite the passage of some 150 years the disease remains incurable, and its medical management is largely supportive. However, the discovery of the DMD gene about 20 years ago has allowed a change in the focus of therapeutic strategy dramatically toward delivery of the missing gene/protein. Indeed, some degree of success has been achieved in preclinical animal studies using such strategies, and gene therapy trials are currently underway in humans. Pharmacological approaches for DMD are also being developed since they can circumvent some of the technical problems associated with gene and cell based therapy. This review explores developments in therapeutic approaches for DMD.

Keywords

Dystrophy Therapy Mdx Utrophin Myostatin 

Abbreviations

BMD

Becker muscular dystrophy

DAPC

Dystrophin-associated protein complex

DMD

Duchenne muscular dystrophy

DRP

Dystrophin-related protein

IGF

Insulin-like growth factor

mdx

Dystrophin-deficient mouse

NMJ

Neuromuscular junction

TGF

Transforming growth factor

Copyright information

© Springer-Verlag 2004

Authors and Affiliations

  • Sasha Bogdanovich
    • 1
  • Kelly J. Perkins
    • 1
  • Thomas O. B. Krag
    • 1
  • Tejvir S. Khurana
    • 1
  1. 1.Department of Physiology and Pennsylvania Muscle InstituteUniversity of Pennsylvania School of MedicinePhiladelphiaUSA

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