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Putting muscle to work for gene therapy

Methods to efficiently transfer and regulate expression of genes in muscle improve the prospects for effective human gene therapy (pages 299–312).

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References

  1. Xiao, X., Li, J. & Samulski, R.J. Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector. J. Viral. 70, 8098–8108 (1996).

    CAS  Google Scholar 

  2. Kessler, P.O. et al. Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein. Proc. Natl. Acod. Sci. USA 93, 14082–14087 (1996).

    Article  CAS  Google Scholar 

  3. Fisher, K.J. et al. Recombinant adeno-associated virus for muscle-directed gene therapy. Nature Med. 3, 306–312 (1997).

    Article  CAS  Google Scholar 

  4. Clark, K.R., Sferra, T.J. & Johnson, P.R. Recombinant adeno-associated virus vectors mediate long-term transgene expression in muscle. Hum. Gene Ther. (in the press).

  5. Bohl, D., Naffakh, N. & Heard, J.M. Long-term control of erythropoietin secretion by doxycycline in mice transplanted with engineered primary myoblasts. Nature Med. 3, 299–305 (1997).

    Article  CAS  Google Scholar 

  6. Wolff, J.A. et al. Direct gene transfer into mouse muscle in vivo. Science 247, 1465–1468 (1990).

    Article  CAS  Google Scholar 

  7. Lynch, C.M., Clowes, M.M., Osborne, W.R.A., Clowes, A.W. & Miller, A.D. Long-term expression of human adenosine deaminase in vascular smooth muscle Cells of rats: A model for gene therapy. Proc. Natl. Acad. Sd. USA 89, 1138–1142 (1992).

    Article  CAS  Google Scholar 

  8. Dai, Y., Roman, M., Naviaux, R.K. & Verma, I.M. Gene therapy via primary myoblasts: long-term expression of factor IX protein following transplantation in vivo. Proc. Natl. Acad. Sci. USA 89, 10892–10895 (1992).

    Article  CAS  Google Scholar 

  9. Osborne, W.R.A., Ramesh, N., Lau, S., Clowes, M.M., Dale, D.C. & Clowes, A.W. Gene therapy for long-term expression of erythropoietin in rats. Proc. Natl. Acad. Sci. USA 92, 8055–8058 (1995).

    Article  CAS  Google Scholar 

  10. Bonham, L., Palmer, T. & Miller, A.D., Prolonged expression of therapeutic levels of human G-CSF in rats following gene transfer to skeletal muscle. Hum. Cene Ther. 7, 1423–1429 (1996).

    Article  CAS  Google Scholar 

  11. Gossen, M. et al. Transcriptional activation by tetracyclines in mammalian Cells. Science 268, 1766–1769 (1995).

    Article  CAS  Google Scholar 

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Miller, A. Putting muscle to work for gene therapy. Nat Med 3, 278–279 (1997). https://doi.org/10.1038/nm0397-278

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