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Nature Medicine  3, 22 - 23 (1997)
doi:10.1038/nm0197-22

Utrophin muscles in on the action

George Karpati

Neuromuscular Research Group and Gene Therapy Unit Montreal Neurological Institute McGill University Montreal, Quebec H3A 2B4, Canada

Overexpressing utrophin in muscles may prove a promising strategy for treating Duchenne muscular dystrophy but many questions remain unanswered.

REFERENCES
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  2. Hoffman, E.P., Brown, R.H.J. & Kunkel, L.N. Dystrophin: the protein product of Duchenne muscular dystrophy locus. Cell 51, 919−928 (1987). | Article | PubMed  | ISI | ChemPort |
  3. Dunnen, J. et al. Topography of the Duchenne muscular dystrophy (DMD) gene: FIGE and cDNA analysis of 194 cases reveals 115 deletions and 13 duplications. Am. J. Hum. Genet. 45, 835−847 (1989). | PubMed  |
  4. Koenig, M. et al. The molecular basis for Duchenne versus Becker muscular dystrophy: Correlation of severity with type of deletion. Am. J. Hum. Genet. 45, 498−506 (1989). | PubMed  | ISI | ChemPort |
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  7. Karpati, G. Recent developments in the biology of dystrophin and related molecules. Curr. Opin. Neurol. Neurosurg. 5, 615−621 (1992). | PubMed  | ISI | ChemPort |
  8. Karpati, G. et al. Myoblast transfer in Duchenne muscular dystrophy. Ann. Neurol. 34, 8−17 (1993). | PubMed  | ISI | ChemPort |
  9. Karpati, G. & Ascadi, G. The potential for gene therapy in neuromuscular diseases. Muscle Nerve 16, 1141−1153 (1993). | PubMed  | ISI | ChemPort |
  10. Tinsley, J.M. et al. Amelioration of the dystrophic phenotype of mdx mice using a truncated utrophin transgene. Nature 384, 349−353 (1996). | Article | PubMed  | ISI | ChemPort |
  11. Blake, D.J., Tinsley, J.M. & Davies, K.E. Utrophin: A structural and functional comparison to dystrophin. Brain Pathol. 6, 37−47 (1996). | PubMed  | ISI | ChemPort |
  12. Karpati, G. et al. Localization and quantitation of the chromosome 6-encoded dystrophin-related protein in normal and pathological human muscle. J. Neuropathol. Exp. Neurol. 52, 119−128 (1993). | PubMed  | ISI | ChemPort |
  13. Matsumara, K., Ervasti, J., Ohlendieck, K., Kahl, S. & Campbell, K.Association of dystrophin-related protein with dystrophin associated proteins in mdx mouse muscle. Nature 60, 588−591 (1992). | Article |
  14. Mizumo, Y., Nonaka, I., Hirai, S. & Ozawa, E. Reciprocal expression of dystrophin and utrophin in muscles of Duchenne muscular dystrophy patients, female DMD carriers and control subjects. J. Neurol. Sci. 119, 43−52 (1993). | Article | PubMed  | ISI | ChemPort |
  15. Vincent, N. et al. Long-term correction of mouse dystrophic degeneration by adenovirus-mediated transfer of a minidystrophin gene. Nature Genet. 5, 130−134 (1993). | PubMed  | ISI | ChemPort |
  16. Lochmüller, H. et al. Transient immunosuppression by FK506 permits a sustained high-level dystrophin expression after adenovirus-mediated dystrophin minigene transfer to skeletal muscles of adult dystrophic (mdx) mice. Gene Ther. 3, 706−716 (1996). | PubMed  | ISI |
  17. Clemens, P.R. et al. In vivo muscle gene transfer of full-length dystrophin with an adenoviral vector that lacks all viral genes. Gene Ther. 3, 965−972 (1996). | PubMed  | ISI | ChemPort |
  18. Cox, G.A. et al. Overexpression of dystrophin in transgenic mdx mice eliminates dystrophic symptoms without toxicity. Nature 364, 725−729 (1993). | Article | PubMed  | ISI | ChemPort |
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ISSN: 1078-8956
EISSN: 1546-170X
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