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Hoffmann, R. A wiki for the life sciences where authorship matters. Nature Genetics (2008)
 

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Long-term correction of mouse dystrophic degeneration by adenovirus-mediated transfer of a minidystrophin gene.

Duchene muscular dystrophy (DMD) is a fatal progressive X-linked muscle disorder, caused by mutations in the dystrophin gene. We have investigated adenovirus-mediated transfer of a dystrophin minigene in a mutant mouse lacking dystrophin, the mdx mouse. We report here that six months after a single intramuscular injection of a recombinant adenovirus containing a human dystrophin minigene, a large number of dystrophin-positive fibres are still detected in the injected muscles. Moreover, although the minigene encodes a truncated protein, its expression is able to protect the fibres efficiently against the degeneration process that affects the dystrophin-deficient mdx myofibres.[1]

References

  1. Long-term correction of mouse dystrophic degeneration by adenovirus-mediated transfer of a minidystrophin gene. Vincent, N., Ragot, T., Gilgenkrantz, H., Couton, D., Chafey, P., Grégoire, A., Briand, P., Kaplan, J.C., Kahn, A., Perricaudet, M. Nat. Genet. (1993) [Pubmed]
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