Skeletal muscle-specific expression of a utrophin transgene rescues utrophin-dystrophin deficient mice

Nature Genetics

Volumn 19, Issue 1, 1998, Pages 79-82

  Rafael, Jill A ^a   Tinsley, Jonathon M ^a   Potter, Allyson C ^a   Deconinck, Anne E ^a   Davies, Kay E ^a  

^a UNIVERSITY OF OXFORD   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

DYSTROPHIN; MUSCLE PROTEIN; UTROPHIN;

ANIMAL TISSUE; ARTICLE; DUCHENNE MUSCULAR DYSTROPHY; GENE EXPRESSION; GENE TARGETING; IMMUNOFLUORESCENCE; MOUSE; NONHUMAN; PHENOTYPE; POLYMERASE CHAIN REACTION; PRIORITY JOURNAL; PROTEIN EXPRESSION; SKELETAL MUSCLE; TRANSGENE;

ANIMALS; CYTOSKELETAL PROTEINS; DYSTROPHIN; FEMALE; GENE EXPRESSION; GENE THERAPY; IMMUNOHISTOCHEMISTRY; MALE; MEMBRANE PROTEINS; MICE; MICE, KNOCKOUT; MUSCLE, SKELETAL; MUSCULAR DYSTROPHY, ANIMAL; TRANSGENES; UTROPHIN;

EID: 0031775820     PISSN: 10614036     EISSN: None     Source Type: Journal    
DOI: 10.1038/ng0598-79     Document Type: Article

References (18)

1. Emery, A.E.H. Duchenne Muscular Dystrophy. (Oxford University Press, Oxford, 1993).
2. Sicinski, P. et al. The molecular basis of muscular dystrophy in the mdx mouse: a point mutation. Science 244, 1578-80 (1989).
3. Bulfield, G., Siller, W.G., Wight, P.A. & Moore, K.J. X chromosome-linked muscular dystrophy (mdx) in the mouse. Proc. Natl. Acad. Sci. USA 81, 1189-92 (1984).
4. Grady, R.M. et al. Skeletal and cardiac myopathies in mice lacking utrophin and dystrophin: a model for Duchenne muscular dystrophy. Cell 90, 729-738 (1997).
5. Deconinck, A.E. et al. Utrophin-dystrophin deficient mice as a model for Duchenne muscular dystrophy. Cell 90, 717-727 (1997).
6. Tinsley, J.M. et al. Amelioration of the dystrophic phenotype of mdx mice using a truncated utrophin transgene. Nature 384, 349-352 (1996).
7. Deconinck, N. et al. Expression of truncated utrophin leads to major functional improvements in dystrophin-deficient muscles of mice. Nature Med. 3, 1216-1221 (1997).
8. Gilbert, R. et al. Efficient utrophin expression following adenovirus gene transfer in dystrophic muscle. Biochem. Biophys. Res. Com. 242, 244-47 (1998).
9. Deconinck, A.E. et al. Postsynaptic abnormalities at the neuromuscular junctions of utrophin-deficient mice. J. Cell Biol. 136, 883-894 (1997).
10. Grady, R.M., Merlie, J.P. & Sanes, J.R. Subtle neuromuscular defects in utrophin-deficient mice. J. Cell Biol. 136, 871-882 (1997).
11. Ohlendieck, K. et al. Dystrophin-related protein is localized to neuromuscular junctions of adult skeletal muscle. Neuron 7, 499-508 (1991).
12. Khurana, T.S. et al. Immunolocalization and developmental expression of dystrophin related protein in skeletal muscle. Neuromuscul. Disord. 1, 185-194 (1991).
13. Ohlendieck, K. & Campbell, K.P. Dystrophin-associated proteins are greatly reduced in skeletal muscle from mdx mice. J. Cell Biol. 115, 1685-1694 (1991).
14. Straub, V. & Campbell, K.P. Muscular dystrophies and the dystrophin-glycoprotein complex. Curr. Opin. Neurol. 10, 168-175 (1997).
15. Tinsley, J.M. & Davies, K.E. Utrophin: A potential replacement for dystrophin? Neuromusc. Disord. 3, 537-539 (1993).
16. Stedman, H.H. et al. The mdx mouse diaphragm reproduces the degenerative changes of Duchenne muscular dystrophy. Nature 352, 535-539 (1991).
17. Dubowitz, V. Muscle disorders in childhood. (W.B. Saunders, London, 1995).
18. Melacini, P. et al. Myocardial involement is very frequent among patients affected with subclinical Becker's muscular dystrophy. Circulation 94, 3168-3175 (1996).