Treatment of the Muscular Dystrophies

As of mid-2002, gene therapy treatment of LGMD was tried in a very small number of patients. These early experiments delivered a functional gene to a very small muscle in the foot and were designed to test the long-term safety and effectiveness of the treatment. Gene therapy for DMD is much more problematic, because of the immense size of the gene and the distribution throughout the body that would be required for effective treatment. Drug treatment with prednisone or other corticosteroids is being used, although at best this provides another six to twelve months of mobility before a wheelchair becomes necessary. There are no effective treatments for myotonic dystrophy as of 2002, although research continues in many laboratories worldwide. see also Gene Therapy; Genetic Testing; Inheritance Patterns; Prenatal Diagnosis; Triplet Repeat Disease.

Jeffrey M. Stajich

Bibliography

Emery, Alan E. H., ed. Neuromuscular Disorders: Clinical and Molecular Genetics. Chichester, U.K.: John Wiley & Sons, 1998.

-. Muscular Dystrophy, The Facts. Oxford, U.K.: Oxford University Press, 2000.

Hoffman, Eric P. "Muscular Dystrophy: Identification and Use of Genes for Diagnostics and Therapeutics." Archives of Pathology and Laboratory Medicine 123 (1999): 1050-1052.

Internet Resource

Muscular Dystrophy Association. <http://www.mdausa.org>.

Mutagen

A mutagen is any substance or agent that can cause a mutation, or change in the sequence or structure of DNA. Mutagens are classified on the basis of their physical nature and the types of damage they do. A mutagen is not the same as a carcinogen. Carcinogens are agents that cause cancer. While many mutagens are carcinogens as well, many others are not. The Ames test is a widely used test to screen chemicals used in foods or medications for mutagenic potential.

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