© 1995 Oxford University Press
RESEARCH-ARTICLE |
Expression of human full-length and minidystrophin in transgenic mdx mice: implications for gene therapy of Duchenne muscular dystrophy
1Department of Veterinary Basic Sciences. The Royal Veterinary College London NW1 OTU 2Department of Experimental Pathology, UMDS, Guy's Hospital London SE1 9RT 3Division of Biochemistry, School of Biological Sciences, Royal Holloway, University of London Surrey TW20 OEX, UK 4Howard Hughes Medical Institute and Department of Physiology and Biophysics, University of Iowa Iowa 52242, USA
*To whom correspondence should be addressed
Received February 16, 1995; Revised May 3, 1995; Accepted May 3, 1995
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 20–30% of endogenous dystrophin levels at the sarcolemma. To the extent that data obtained in mouse studies are applicable to DMD, the virtual elimination of morphological and biochemical abnormalities in the mdx mouse supports the use of this cDNA in somatic gene therapy protocols for DMD.
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