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Therapeutics for Duchenne muscular dystrophy: current approaches and future directions

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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.

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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

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Acknowledgements

We thank Dr. Carsten Bönneman (University of Pennsylvania) and Dr. Roland Roberts (Guy’s Hospital, London) for insightful comments. This research was supported by grants from the Association Francaise contre Les Myopathies (France), Muscular Dystrophy Association (USA), Duchenne Parents Project (The Netherlands), and RO1 AR 048871 from the National Institutes of Health to T.S.K.

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  1. Department of Physiology and Pennsylvania Muscle Institute, University of Pennsylvania School of Medicine, 3700 Hamilton Walk, Richards A-601, Philadelphia, PA 19104-6085, USA

    Sasha Bogdanovich, Kelly J. Perkins, Thomas O. B. Krag & Tejvir S. Khurana

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  1. Sasha Bogdanovich
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  2. Kelly J. Perkins
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  3. Thomas O. B. Krag
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Correspondence to Tejvir S. Khurana.

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S. Bogdanovich, K.J. Perkins contributed equally to this work

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Bogdanovich, S., Perkins, K.J., Krag, T.O.B. et al. Therapeutics for Duchenne muscular dystrophy: current approaches and future directions. J Mol Med 82, 102–115 (2004). https://doi.org/10.1007/s00109-003-0484-1

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