Genetic Correction of Duchenne Muscular Dystrophy using Engineered Nucleases
<p>Duchenne muscular dystrophy (DMD) is a severe hereditary disorder caused by a loss of dystrophin, an essential musculoskeletal protein. Decades of promising research have yielded only modest gains in survival and quality of life for these patients and there have been no approved gene therap...
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2014
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| Online Access: | http://hdl.handle.net/10161/8769 |