Amelioration of the dystrophic phenotype of mdx mice using a truncated utrophin transgene

DUCHENNE muscular dystrophy (DMD) is a severe, progressive muscle-wasting disease that causes cardiac or respiratory failure 1,2 and results in death at about 20 years of age. Replacement of the missing protein, dystrophin, using myoblast transfer in humans or viral/liposomal delivery in the mouse D...

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Veröffentlicht in:	Nature (London) 1996-11, Vol.384 (6607), p.349-353
Hauptverfasser:	Tinsley, Jonathon M., Potter, Allyson C., Phelps, Steven R., Fisher, Rosie, Trickett, Jeffrey I., Davies, Kay E.
Format:	Artikel
Sprache:	eng
Schlagworte:	Animals Biological and medical sciences Creatine Kinase - blood Cytoskeletal Proteins - genetics Cytoskeletal Proteins - therapeutic use Diseases of striated muscles. Neuromuscular diseases Dystrophin - genetics Dystrophy Female Gene Expression Regulation Genetic Therapy Humanities and Social Sciences Humans letter Male Medical sciences Membrane Proteins Mice Mice, Inbred C57BL Mice, Inbred CBA Mice, Mutant Strains Mice, Transgenic multidisciplinary Muscles Muscles - metabolism Muscular Dystrophies - genetics Muscular Dystrophies - therapy Neurology Pathology Science Science (multidisciplinary) Transgenes Up-Regulation Utrophin
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