Muscle-based gene therapy: Realistic possibilities for the future

Molecular Medicine Today

Volumn 2, Issue 4, 1996, Pages 166-172

  Svensson, Eric C ^a   Tripathy, Sandeep K ^a   Leiden, Jeffrey M ^b  

^a UNIVERSITY OF CHICAGO   (United States)

^b UNIVERSITY OF CHICAGO   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

DNA; PLASMA PROTEIN; RECOMBINANT VACCINE;

ANIMAL; CELL TRANSPLANTATION; CYTOLOGY; GENE THERAPY; GENE VECTOR; GENETICS; HUMAN; IMMUNOLOGY; INBORN ERROR OF METABOLISM; METHODOLOGY; MUSCULAR DYSTROPHY; REVIEW; SKELETAL MUSCLE;

ANIMALS; BLOOD PROTEINS; CELL TRANSPLANTATION; DNA; GENE THERAPY; GENETIC VECTORS; HUMANS; METABOLISM, INBORN ERRORS; MUSCLE, SKELETAL; MUSCULAR DYSTROPHIES; VACCINES, SYNTHETIC;

EID: 0030117962     PISSN: 13574310     EISSN: None     Source Type: Journal    
DOI: 10.1016/1357-4310(96)88792-2     Document Type: Review

References (40)

1. Mauro, A. (1961) Satellite cell of skeletal muscle fibers, J. Biophys. Biochem. Cytol 9, 493-495
2. Rando, T.A. and Blau, H.M. (1994) Primary mouse myoblast purification, characterization, and transplantation for cell-mediated gene therapy, J. Cell Biol. 125, 1275-1287
3. Blau, H.M., Dhawan, J. and Pavlath, G.K. (1993) Myoblasts in pattern formation and gene therapy, Trends Genet. 9, 269-274
4. Barr, E. and Leiden, J.M. (1991) Systemic delivery of recombinant proteins by genetically modified myoblasts, Science 254, 1507-1509
5. Dhawan, J. et al. (1991) Systemic delivery of human growth hormone by injection of genetically engineered myoblasts, Science 254, 1509-1512
6. Osborne, W.A.R. et al. (1995) Gene therapy for long-term expression of erythropoietin in rats, Proc. Natl Acad. Sci. USA 92, 8055-8058
7. Partridge, T.A. et al. (1989) Conversion of mdx myofibers from dystrophin-negative to -positive by injection of normal myoblasts, Nature 337, 176-179
8. Wolff, J.A. et al. (1990) Direct gene transfer into mouse muscle in vivo, Science 247, 1465-1468
9. Ulmer, J.B. et al. (1993) Heterologous protection against influenza by injection of DNA encoding a viral protein, Science 259, 1745-1749
10. Yao, S.N. and Kurachi, K. (1992) Expression of human factor IX in mice after injection of genetically modified myoblasts, Proc. Natl Acad. Sci. USA 89, 3357-3361
11. Dai, Y., Roman, M., Naviaux, R.K. and Verma, I.M. (1992) Gene therapy via primary myoblasts: long-term expression of factor IX protein following transplantation in vivo, Proc. Natl Acad. Sci. USA 89, 10892-10895
12. Mendell, J.R. et al. (1995) Myoblast transfer in the treatment of Duchenne's muscular dystrophy, New Engl. J. Med. 333, 832-838
13. Morgan, J.E. (1994) Cell and gene therapy in Duchenne muscular dystrophy, Hum. Gene Ther. 5, 165-173
14. Ragot, T. et al. (1993) Efficient adenovirus-mediated transfer of a human minidystrophin gene to skeletal muscle of mdx mice, Nature 361, 647-650
15. Tripathy, S.K. et al. (1994) Stable delivery of physiologic levels of recombinant erythropoietin to the systemic circulation by intramuscular injection of replication-defective adenovirus, Proc. Natl Acad. Sci. USA 91, 11557-11561
16. Dai, Y. et al. (1995) Cellular and humoral immune responses to adenoviral vectors containing factor IX gene: tolerization of factor IX and vector antigens allows for long-term expression, Proc. Natl Acad. Sci. USA 92, 1401-1405
17. Wolff, J.A. et al. (1992) Long-term persistence of plasmid DNA and foreign gene expression in mouse muscle, Hum. Mol. Genet. 1, 363-369
18. Acsadi, G. et al. (1991) Human dystrophin expression in mdx mice after intramuscular injection of DNA constructs, Nature 352, 815-818
19. Dunckley, M.G., Wells, D.J., Walsh, F.S. and Dickson, G. (1993) Direct retroviral-mediated transfer of a dystrophin minigene into mdx mouse muscle in vivo, Hum. Mol. Genet. 2, 717-723
20. Huard, J. et al. (1994) High efficiency of muscle regeneration after human myoblast clone transplantation in SCID mice, J. Clin. Invest. 93, 586-599
21. Pavlath, G.K., Rando, T.A. and Blau, H.M. (1994) Transient immunosuppressive treatment leads to long-term retention of allogeneic myoblasts in hybrid myofibers, J. Cell Biol. 127, 1923-1932
22. Manthorpe, M. et al. (1993) Gene therapy by intramuscular injection of plasmid DNA: studies on firefly luciferase gene expression in mice, Hum. Gene Ther. 4, 419-431
23. Davis, H.L., Whalen, R.G. and Demeneix, B.A. (1993) Direct gene transfer into skeletal muscle in vivo: factors affecting efficiency of transfer and stability of expression, Hum. Gene Ther. 4, 151-159
24. Yang, Y. et al. (1994) Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy, Proc. Natl Acad. Sci. USA 91, 4407-4411
25. Flotte, T.R. and Carter, B.J. (1995) Adeno-associated virus vectors for gene therapy, Gene Ther. 2, 357-362
26. Thierry, A. R. et al. (1995) Systemic gene therapy: biodistribution and long-term expression of a transgene in mice, Proc. Natl Acad. Sci. USA 92, 9742-9746
27. Vincent, N. et al. (1993) Long-term correction of mouse dystrophic degeneration by adenovirus-mediated transfer of a minidystrophin gene, Nat. Genet. 5, 130-134
28. Dwarki, V.J. et al. (1995) Gene therapy for hemophilia A: production of therapeutic levels of human factor VIII in vivo in mice, Proc. Natl Acad. Sci. USA 92, 1023-1027
29. Newgard, C.B. (1994) Cellular engineering and gene therapy strategies for insulin replacement in diabetes, Diabetes 43, 341-350
30. Hamamori, Y., Samal, B., Tian, J. and Kedes, L. (1995) Myoblast transfer of human erythropoietin gene in a mouse model of renal failure, J. Clin. Invest. 95, 1808-1813
31. 1-antitrypsin in mice after direct gene delivery in vivo, Hum. Gene Ther. 3, 641-647
32. 1-antitrypsin gene to the lung epithelium in vivo, Science 252, 431-434
33. Salvetti, A., Heard, J.M. and Danos, O. (1995) Gene therapy of lysosomal storage disorders, Br. Med. Bull. 51, 106-122
34. Braun, S.E. et al. (1993) Metabolic correction and cross-correction of mucopolysaccharidosis type II (Hunter syndrome) by retroviral-mediated gene transfer and expression of human iduronate-2-sulfatase, Proc. Natl Acad. Sci. USA 90, 11830-11834
35. Kohn, D.B. et al. (1991) Toward gene therapy for Gaucher disease, Hum. Gene Ther. 2, 101-105
36. Wolfe, J.H. et al. (1992) Reversal of pathology in murine mucopolysaccharidosis type VII by somatic cell gene transfer, Nature 360, 749-753
37. Taylor, R.M. and Wolfe, J.H. (1994) Cross-correction of β-glucuronidase deficiency by retroviral vector-mediated gene transfer, Exp. Cell Res. 214, 606-613
38. + T lymphocytes, J. Immunol. 155, 259-265
39. Wang, B. et al. (1993) Gene inoculation generates immune responses against human immunodeficiency virus type 1, Proc. Natl Acad. Sci. USA 90, 4156-4160
40. Davis, H.L., Michel, M.L. and Whalen, R.G. (1993) DNA-based immunization induces continuous secretion of hepatitis B surface antigen and high levels of circulating antibody, Hum. Mol. Genet. 2, 1847-1851