Article
Progress toward Gene Therapy for Duchenne Muscular Dystrophy.
Molecular therapy : the journal of the American Society of Gene Therapy - 3 May 2017
Chamberlain Joel R, Chamberlain Jeffrey S
Abstract excerpt
Duchenne muscular dystrophy (DMD) has been a major target for gene therapy development for nearly 30 years. DMD is among the most common genetic diseases, and isolation of the defective gene (DMD, or dystrophin) was a landmark discovery, as it was the first time a human disease gene had been cloned without knowledge of the protein product. Despite tremendous obstacles, including the enormous size of the gene and...
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