Article
Genetic therapeutic approaches for Duchenne muscular dystrophy.
Human gene therapy - 1 Jul 2012
Foster Helen, Popplewell Linda, Dickson George
Abstract excerpt
Despite an expansive wealth of research following the discovery of the DMD gene 25 years ago, there is still no curative treatment for Duchenne muscular dystrophy. However, there are currently many promising lines of research, including cell-based therapies and pharmacological reagents to upregulate dystrophin via readthrough of nonsense mutations or by upregulation of the dystrophin homolog utrophin. Here we...
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