Nature Medicine
3, 22 - 23 (1997)
doi:10.1038/nm0197-22
Utrophin muscles in on the actionGeorge KarpatiNeuromuscular 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
- Koenig, M. et al. Complete cloning of the Duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals. Cell 50, 509−517 (1987). | Article | PubMed | ISI | ChemPort |
- 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 |
- 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 |
- 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 |
- Sunada, Y. & Campbell, K. Dystrophin-glycoprotein complex: Molecular organization and critical roles in skeletal muscle. Curr. Opin. Neurol. 8, 379−384 (1995). | PubMed | ISI | ChemPort |
- Ozawa, E. et al. Dystrophin-associated proteins in muscular dystrophy. Hum. Mol. Genet. 4, 1711−1716 (1995). | PubMed | ISI | ChemPort |
- Karpati, G. Recent developments in the biology of dystrophin and related molecules. Curr. Opin. Neurol. Neurosurg. 5, 615−621 (1992). | PubMed | ISI | ChemPort |
- Karpati, G. et al. Myoblast transfer in Duchenne muscular dystrophy. Ann. Neurol. 34, 8−17 (1993). | PubMed | ISI | ChemPort |
- Karpati, G. & Ascadi, G. The potential for gene therapy in neuromuscular diseases. Muscle Nerve 16, 1141−1153 (1993). | PubMed | ISI | ChemPort |
- 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 |
- 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 |
- 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 |
- 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 |
- 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 |
- 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 |
- 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 |
- 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 |
- 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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