Article
The Pathogenesis and Therapy of Muscular Dystrophies.
Annual review of genomics and human genetics - 1 Jan 2015
Guiraud Simon, Aartsma-Rus Annemieke, Vieira Natassia M, Davies Kay E, van Ommen Gert-Jan B, Kunkel Louis M
Abstract excerpt
Current molecular genomic approaches to human genetic disorders have led to an explosion in the identification of the genes and their encoded proteins responsible for these disorders. The identification of the gene altered by mutations in Duchenne and Becker muscular dystrophy was one of the earliest examples of this paradigm. The nearly 30 years of research partly outlined here exemplifies the road that similar...
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