Long–term correction of mouse dystrophic degeneration by adenovirus–mediated transfer of a minidystrophin gene (original) (raw)

References

  1. Emery, A.E.H. Duchenne muscular Dystrophy (Oxford University Press, Oxford, 1988).
    Google Scholar
  2. Hoffman, E.P., Brown, J., R.H. & Kunkel, L.M. Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell 51, 919–928 (1987).
    Article CAS Google Scholar
  3. Butfield, G., Siller, W.G., Wight, P.A.L. & Moore, K.J. X chromosome-linked muscular dystrophy (mdx) in the mouse. Proc. natn Acad. Sci. U.S.A. 81, 1189–1192 (1984).
    Article Google Scholar
  4. Sicinski, P., Geng, Y., Ryder-Cook, A., Barnard, E., Darlinson, M. & Barnard, P. The molecular basis of muscular dystrophy in the mdx mouse: a point mutation. Science 244, 1578–1580 (1989).
    Article CAS Google Scholar
  5. McGeachie, J.K., Grounds, M.D., Partridge, T.A. & Morgan, J.E. Replication of myogenic cells with age, and myogenesis after experimental injury in mdx mouse muscle: quantitative autoradiographic studies. Duchenne Muscular Dystrophy: Animal models and genetic manipulation. 189–201 (Raven Press, New York, 1992).
    Google Scholar
  6. Wells, D.J. et al. Human dystrophin expression corrects the myopathic phenotype in transgenic mdx mice. Hum. molec. Genet. 1, 35–40 (1992).
    Article CAS Google Scholar
  7. England, S. et al. Very mild muscular dystrophy associated with the deletion of 46% of dystrophin. Nature 343, 180–182 (1990).
    Article CAS Google Scholar
  8. Ragot, T. et al. Efficient adenovirus-mediated transfer of a human minidystrophin gene to skeletal muscle of mdx mice muscle. Nature 361, 647–650 (1993).
    Article CAS Google Scholar
  9. Stratford-Perricaudet, L.D., Makeh, I., Pemcaudet, M. & Briand, P. Widespread long-term gene transfer to mouse skeletal muscles and heart via an adenovirus vector. J. clin. Invest 90, 626–630, (1992).
    Article CAS Google Scholar
  10. Cartaud, A. et al. Localization of dystrophin and dystrophin-related protein at the electromotor synapse and neuromuscular junction in Torpedo Marmorata. Neurosci. 48, 995–1003 (1992).
    Article CAS Google Scholar
  11. Hoffman, E.P., Morgan, J., Watkins, S.C. & Partridge, T.A. Somatic reversion/ suppression of the mouse mdx phenotype in vivo. J. Neurol. Sci. 99, 9–25 (1990).
    Article CAS Google Scholar
  12. Akli, S. et al. Transfer of a foreign gene into the brain using adenovirus vector. Nature Genet. 3, 224–228 (1993).
    Article CAS Google Scholar
  13. Partridge, T.E., Morgan, J.E., Coulton, J.R., Hoffman, E.P. & Kunkel, L.M. Conversion of mdx myofibres from dystrophin-negative to -positive by injection of normal myoblasts. Nature 337, 176–179 (1989).
    Article CAS Google Scholar
  14. Morgan, J., Hoffman, E.P. & Partridge, T.A. Normal myogenic cells from newborn mice restore normal histology to degenerating muscle of the mdx mouse. J. cell Biol. 111, 2437–2449 (1990).
    Article CAS Google Scholar
  15. Acsadi, G. et al. Human dystrophin expression in mdx mice after injection of DNA constructs. Nature 352, 815–818 (1991).
    Article CAS Google Scholar
  16. Dunckley, M.G., Wells, D.J., Walsh, F.S. & Dickson, G. Direct retroviral-mediated transfer of a dystrophin minigene into mdx mouse muscle in vivo. Hum. molec. Genet. 2, 717–723 (1993).
    Article CAS Google Scholar
  17. Huard, J. et al. Human myoblasts transplantation between immunohistocompatible donors and recipients produces immune reactions. Transpl. Proc. 24, 3049–3051 (1992).
    CAS Google Scholar
  18. Dubowitz, V. Myoblast transfer in muscular dystrophy: panacea or pie in the sky?. Neuromusc. Disord. 2, 305–310 (1992).
    Article CAS Google Scholar
  19. Jiao, S. et al. Direct gene transfer into nonhuman primate myofibers in vivo. Hum. gene Ther. 3, 21–33 (1992).
    Article CAS Google Scholar
  20. Davis, H.L., Whalen, R.G. & Demeneix, B.A. Direct gene transfer into skeletal muscle in vivo: factors affecting efficiency of transfer and stability of expression. Hum. gene Ther. 4, 151–159 (1993).
    Article Google Scholar
  21. Cooper, B.J. et al. The homologue of Duchenne locus is defective in X-linked muscular dystrophy of dogs. Nature 334, 154–156 (1988).
    Article CAS Google Scholar
  22. Gluzman, Y., Reichl, H. & Solnick, D. Helper-free adenovirus type 5 vector. In Eukaryotic Viral Vectors (ed. Gluzman, Y.) 187–192 (Cold Spring Harbor Laboratory, New York, 1982).
    Google Scholar
  23. Shenk, T. & Williams, J. Genetic analysis of adenovirus. in Curr. Top. Microbiol. Immun. (ed Doerfler, W.) 111, 1–39 (Springer-Verlag, Berlin, 1984).
    Google Scholar
  24. Graham, F.L., Smiley, J., Russel, W.C. & Nairn, R. Characteristics of a human cell line transformed by DNA from human adenovirus type 5. J. gen. Virol. 36, 59–72 (1977).
    Article CAS Google Scholar
  25. Hugnot, J.P. et al. Novel products of the dystrophin gene: a distal transcript initiated from a unique alternative first exon encoding for a 75 kDa protein widely distributed in non-muscle tissues. Proc. natn Acad. Sci. U.S.A. 89, 7506–7510 (1992).
    Article CAS Google Scholar

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