Putting muscle to work for gene therapy

Nature Medicine

Volumn 3, Issue 3, 1997, Pages 278-279

  Dusty Miller, A ^a  

^a Fred Hutchinson Cancer Research Center   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; HUMAN; MUSCLE CONTRACTION; MUSCULAR DYSTROPHY; NOTE; PRIORITY JOURNAL;

GENE EXPRESSION REGULATION; GENE TARGETING; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS; MUSCLE, SKELETAL;

EID: 16944364154     PISSN: 10788956     EISSN: None     Source Type: Journal    
DOI: 10.1038/nm0397-278     Document Type: Note

References (11)

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. Virol. 70, 8098-8108 (1996).
2. Kessler, P.D. et al. Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein. Proc. Natl. Acad. Sci. USA 93, 14082-14087 (1996).
3. Fisher K.J. et al. Recombinant adeno-associated virus for muscle-directed gene therapy. Nature Med. 3, 306-312 (1997).
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).
6. Wolff, J.A. et al. Direct gene transfer into mouse muscle in vivo. Science 247, 1465-1468 (1990).
7. Lynch, CM., 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. Sci. USA 89, 1138-1142 (1992).
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).
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).
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. Gene Ther. 7, 423-1429 (1996).
11. Gossen, M. et al. Transcriptional activation by tetracyclines in mammalian cells. Science 268, 1766-1769 (1995).