Stable restoration of the sarcoglycan complex in dystrophic muscle perfused with histamine and a recombinant adeno-associated viral vector (original) (raw)

References

  1. Bonnemann, C., McNally, E. & Kunkel, L. Beyond dystrophin: current progress in the muscular dystrophies. Curr. Opin. Pediatr. 8, 569 –582 (1996).
    Article CAS Google Scholar
  2. Homburger, F., Baker, J., Nixon, C. & Whitney, R. Primary, generalized polymyopathy and necrosis in an inbred line of Syrian hamsters. Med. Exp. 6, 339–345 ( 1962).
    Google Scholar
  3. Roberds, S. et al. Disruption of the dystrophin-glycoprotein complex in the cardiomyopathyic hamster. J. Biol. Chem. 268, 11496– 11499 (1993).
    CAS PubMed Google Scholar
  4. Nigro, V. et al. Identification of the Syrian hamster cardiomyopathy gene. Hum. Mol. Genet. 6, 601–607 (1997).
    Article CAS Google Scholar
  5. Sakamoto, A. et al. Both hypertrophic and dilated cardiomyopathies are caused by mutation of the same gene, delta sarcoglycan, in hamster: an animal model of disrupted dystrophin-associated glycoprotein complex. Proc. Natl. Acad. Sci. USA 94, 13873–13878 (1997).
    Article CAS Google Scholar
  6. Worton, R. Muscular dystrophies: diseases of the dystrophin-glycoprotein complex. Science 270, 755–6 ( 1995).
    Article CAS Google Scholar
  7. Fisher, K. et al. Recombinant adeno-associated virus for muscle directed gene therapy. Nature Med. 3, 306–312 (1997).
    Article CAS Google Scholar
  8. Homburger, F., Baker, J., Wilgram, G., Caulfield, J. & Nixon, C. Hereditary dystrophy-like myopathy. The histopathology of hereditary dystrophy-like myopathy in Syrian hamsters. Arch. Pathol. 81, 302–7 ( 1966).
    CAS PubMed Google Scholar
  9. Cox, G. et al. Overexpression of dystrophin in transgenic mdx mice eliminates dystrophic symptoms without toxicity. Nature 364, 725–29 (1993).
    Article CAS Google Scholar
  10. Ragot, T. et al. Efficient adenovirus-mediated transfer of a human minidystrophin gene to skeletal muscle of mdx mice. Nature 361, 647–650 (1993).
    Article CAS Google Scholar
  11. Holt, K. et al. Functional rescue of the sarcoglycan complex in the BIO 14.6 hamster using delta-sarcoglygan gene transfer. Mol. Cell 1, 841–848 (1998).
    Article CAS Google Scholar
  12. Cox, G.A., Sunada, Y., Campbell, K.P. & Chamberlain, J.S. Dp71 can restore the dystrophin-associated glycoprotein complex in muscle but fails to prevent dystrophy. Nature Genet. 8, 333–339 (1994).
    Article CAS Google Scholar
  13. Petrof, B.J., Shrager, J.B., Stedman, H.H., Kelly, A.M. & Sweeney, H.L. Dystrophin protects the sarcolemma from stresses developed during muscle contraction. Proc. Natl. Acad. Sci. 90, 3710–14 ( 1993).
    Article CAS Google Scholar
  14. Straub, V., Rafael, J., Chamberlain, J. & Campbell, K. Animal models for muscular dystrophy show different patterns of sarcolemmal disruption. J. Cell Biol. 139, 375– 385 (1997).
    Article CAS Google Scholar
  15. Deconinck, N., Ragot, T., Marechal, G., Perricaudet, M. & Gillis, J. Functional protection of dystrophic mouse (mdx) muscles after adenovirus-mediated transfer of a dystrophin minigene. Proc. Natl. Acad. Sci. USA 93, 3570–3574 (1996).
    Article CAS Google Scholar
  16. DeMatteo, R., Chu, G., Chang, E., Barker, C. & Markmann, J. Long-lasting adenovirus transgene expression in mice through neonatal intrathymic tolerance induction without the use of immunosuppression. J. Virol. 71, 5330–5 (1997).
    CAS PubMed PubMed Central Google Scholar
  17. Nielsen, H. Histamine-2 receptor antagonists as immunomodulators: new therapeutic views? Ann. Med. 28, 107–13 (1996).
    Article CAS Google Scholar
  18. Stedman, H.H. et al. Nebulin cDNAs detect a 25-kilobase transcript in skeletal muscle and localize to human chromosome 2. Genomics 2, 1–7 (1988).
    Article CAS Google Scholar
  19. Samulski, R., Chang, L. & Shenk, T. A recombinant plasmid–from which an infectious adeno-associated virus genome can be excised in vitro and its use to study viral replication. J. Virol. 61, 3096-3101 (1987).
    CAS PubMed PubMed Central Google Scholar
  20. Sanes, J., Rubenstein, J. & Nicolas, J. Use of a recombinant retrovirus to study post-implantation cell lineage in mouse embryos. EMBO J. 5, 3133–3142 (1986).
    Article CAS Google Scholar
  21. Brumback, R.A. & and Leech, R.W. in Color Atlas of Muscle Histochemistry, 13;21 (PSG, Littleton, Massachusetts, 1984).
    Google Scholar

Download references