Article
Expression of human full-length and minidystrophin in transgenic mdx mice: implications for gene therapy of Duchenne muscular dystrophy.
Human molecular genetics - 1 Aug 1995
Wells D J, Wells K E, Asante E A, Turner G, Sunada Y, Campbell K P, Walsh F S, Dickson G
Abstract excerpt
Duchenne muscular dystrophy (DMD) is a lethal X-linked recessive disorder with a high spontaneous mutation rate and no effective treatment, hence development of genetic based therapies is an important goal. We report that expression of a recombinant human minidystrophin cDNA, compatible with current viral vectors, can significantly reduce the myopathic phenotype in transgenic mdx mice, even when expressed at only...
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