The fate of individual myoblasts after transplantation into muscles of DMD patients

Nature Medicine

Volumn 3, Issue 9, 1997, Pages 970-974

  Gussoni, Emanuela ^a   Blau, Helen M ^b   Kunkel, Louis M ^a  

^a BOSTON CHILDREN S HOSPITAL   (United States)

^b STANFORD UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

DYSTROPHIN;

ARTICLE; CELL TRANSPLANTATION; CLINICAL ARTICLE; DUCHENNE MUSCULAR DYSTROPHY; FLUORESCENCE IN SITU HYBRIDIZATION; GENE MUTATION; GENE THERAPY; HUMAN; HUMAN TISSUE; MUSCLE ATROPHY; MUSCLE BIOPSY; MUSCLE WEAKNESS; MYOBLAST; ORGAN DONOR; PRIORITY JOURNAL; X CHROMOSOME RECESSIVE INHERITANCE;

CELL COUNT; CELL FUSION; CELL NUCLEUS; CELL TRANSPLANTATION; DYSTROPHIN; GENE EXPRESSION; HUMANS; IMMUNOHISTOCHEMISTRY; IN SITU HYBRIDIZATION, FLUORESCENCE; MALE; MUSCLES; MUSCULAR DYSTROPHIES; T-LYMPHOCYTES; TIME FACTORS; TISSUE DONORS;

EID: 0030865245     PISSN: 10788956     EISSN: None     Source Type: Journal    
DOI: 10.1038/nm0997-970     Document Type: Article

References (57)

1. Emery, A. Duchenne Muscular Dystrophy, Vol. 1, 317 pp. (Oxford Univ. Press, New York, 1988).
2. Engel, A.G., Yamamoto, M. & Fischbeck, K.H. in Myology. (eds. Engel, A.G. & Franzini-Armstrong, C.) 1130-1188 (McGraw-Hill, New York, 1994).
3. Monaco, A.P. et al. Isolation of candidate cDNAs for portions of the Duchenne muscular dystrophy gene. Nature 323, 646-650 (1986).
4. Burghes, A.H.M. et al. A cDNA clone from the Duchenne/Becker muscular dystrophy gene. Nature 328, 434-437 (1987).
5. Hoffman, E.P., Brown, R.H. & Kunkel, L.M. Dystrophin: The protein product of the Duchenne muscular dystrophy locus. Cell 51, 919-928 (1987).
6. Nudel, U., Robzyk, K. & Yaffe, D. Expression of the putative Duchenne muscular dystrophy gene in differentiated myogenic cell cultures and in the brain. Nature 331, 635-638 (1988).
7. Lidov, H.G.W., Byers, T.J., Watkins, S.C. & Kunkel, L.M. Localization of dystrophin to postsynaptic regions of central nervous system cortical neurons. Nature 348, 725-728 (1990).
8. Byers, T.J., Kunkel, L.M. & Watkins, S.C. The subcellular distribution of dystrophin in mouse skeletal, cardiac and smooth muscle. J. Cell Biol. 115, 411-421 (1991).
9. Hoffman, E.P. et al. Dystrophin characterization in muscle biopsies from Duchenne and Becker muscular dystrophy patients. N. Engl. J. Med. 318, 1363-1368 (1988).
10. Bulfield, C., Siller, W.G., Wight, P.A.L. & Moore, K.J. X chromosome-linked muscular dystrophy (mdx) in the mouse. Proc. Natl. Acad. Sci. USA 81, 1189-1192 (1984).
11. Sicinski, P. et al. The molecular basis of muscular dystrophy in the mdx mouse: A point mutation. Science 244, 1578-1580 (1989).
12. Cox, G.A. et al. Overexpression of dystrophin in transgenic mdx mice eliminates dystrophic symptoms without toxicity. Nature 364, 725-729 (1993).
13. Phelps, S.F. et al. Expression of full-length and truncated dystrophin mini-genes in transgenic mdx mice. Hum. Mol. Genet. 4, 1251-1258 (1995).
14. Wells, D.J. et al. Expression of human full-length and minidystrophin in transgenic mdx mice: Implications for gene therapy of Duchenne muscular dystrophy. Hum. Mol. Genet. 4, 1245-1250 (1995).
15. Tinsley, J.M. et al. Amelioration of the dystrophic phenotype of mdx mice using a truncated utrophin transgene. Nature 384, 349-353 (1996).
16. Acsadi, G. et al. Human dystrophin expression in mdx mice after intramuscular injections of DNA constructs. Nature 352, 815-818 (1991).
17. 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. Mol. Genet. 2, 717-723 (1993).
18. Ragot, T. et al. Efficient adenovirus-mediated transfer of a human minidystrophin gene to skeletal muscle of mdx mice. Nature 361, 647-650 (1993).
19. Vincent, N. et al. Long-term correction of mouse dystrophic degeneration by adenovirus-mediated transfer of a minidystrophin gene. Nature Genet. 5, 130-134 (1993).
20. Kochanek, S. et al. A new adenoviral vector: Replacement of all viral coding sequences with 28 kb of DNA independently expressing both full-length dystrophin and β-galactosidase. Proc. Natl. Acad. Sci. USA 93, 5731-5736 (1996).
21. Kumar-Singh, R. & Chamberlain, J.S. Encapsidated adenovirus minichromosomes allow delivery and expression of a 14Kb dystrophin cDNA to muscle cells. Hum. Mol. Genet. 5, 913-921 (1996).
22. Karpati, C. et al. Dystrophin is expressed in mdx skeletal muscle fibers after normal myoblast implantation. Am. J. Pathol. 135, 27-32 (1989).
23. Griggs, R.C. & Karpati, G (eds.). Myoblast Transfer Therapy. 316 pp. (Plenum, New York, 1990).
24. Partridge, T.A. Myoblast transfer: A possible therapy for inherited myopathies? [invited review]. Muscle Nerve 14, 197-212 (1991).
25. Fassati, A., Wells, D.J., Walsh, F.S. & Dickson, G. Efficiency of in vivo gene transfer using murine retroviral vectors is strain-dependent in mice. Hum. Gene Therapy 6, 1177-1183 (1995).
26. Guerette, B., Asselin, I., Vilquin, J.T., Roy, R. & Tremblay, J.P. Lymphocyte infiltration following allo-and xenomyoblast transplantation in mdx mice. Muscle Nerve 18, 39-51 (1995).
27. Vilquin, J.-T. et al. FK506 Immunosuppression to control the immune reactions triggered by first-generation adenovirus-mediated gene transfer. Hum. Gene Ther. 6, 1391-1401 (1995).
28. Yang, Y., Haecker, S.E., Su, Q. & Wilson, J.M. Immunology of gene therapy with adenoviral vectors in mouse skeletal muscle. Hum. Mol. Genet. 5, 1703-1712 (1996).
29. Mauro, A. Satellite cells of skeletal muscle. J. Biophys. Biochem. Cytol. 9, 493-495 (1961).
30. Konigsberg, I. The differentiation of cross-striated myofibrils in short-term cell culture. Exp. Cell Res. 21, 414-420 (1960).
31. Richler, C. & Yaffe, D. The in vitro cultivation and differentiation capacities of myogenic cell lines. Dev. Biol. 23, 1-22 (1970).
32. Hauschka, S.D. Clonal analysis of vertebrate myogenesis. II. Environmental influences upon human muscle differentiation. Dev. Biol. 37, 329-344 (1974).
33. Blau, H.M. & Webster, C. Isolation and characterization of human muscle cells. Proc. Natl. Acad. Sci. USA 78, 5623-5627 (1981).
34. Rando, T.A. & Blau, H.M. Primary mouse myoblast purification, characterization, and transplantation for cell-mediated gene therapy. J. Cell Biol. 125, 1275-1287 (1994).
35. Barr, E. & Leiden, J. Systemic delivery of recombinant proteins by genetically modified myoblasts. Science 254, 1507-1509 (1991).
36. Dhawan, J. et al. Systemic delivery of human growth hormone by injection of genetically engineered myoblasts. Science 254, 1509-1512 (1991).
37. Law, P.K. et al. Dystrophin production induced by myoblast transfer therapy in Duchenne muscular dystrophy. Lancet 336, 114-115 (1990).
38. Gussoni, E. et al. Normal dystrophin transcripts detected in Duchenne muscular dystrophy patients after myoblast transplantation. Nature 356, 435-438 (1992).
39. Huard, J. et al. Human myoblast transplantation: Preliminary results of 4 cases. Muscle Nerve 15, 550-560 (1992).
40. Karpati, C. et al. Myoblast transfer in Duchenne muscular dystrophy. Ann. Neurol. 34, 8-17 (1993).
41. Mendell, J.R. et al. Myoblast transfer in the treatment of Duchenne's muscular dystrophy. N. Engl. J. Med. 333, 832-838 (1995).
42. Morandi, L. et al. Lack of mRNA and dystrophin expression in DMD patients three months after myoblast transfer. Neuromusc. Disord. 5, 291-295 (1995).
43. Gussoni, E. et al. A method to co-detect introduced genes and their products in gene therapy protocols. Nature Biotechnol. 14, 1012-1016 (1996).
44. Schultz, E., Jaryszack, D.L. & Valliere, C.R. Response of satellite cells to focal skeletal muscle injury. Muscle Nerve 8, 217-222 (1985).
45. Grounds, M. The proliferation and fusion of myoblasts in vivo. Adv. Exp. Med. Biol. 280, 101-104 (1990).
46. Hoffman, E.P., Morgan, J.E., Watkins, S.C. & Partridge, T.A. Somatic reversion/ suppression of the mouse mdx phenotype in vivo. J. Neuroi. Sci. 99, 9-25 (1990).
47. Fanin, M. et al. Dystrophin-positive fibers in Duchenne dystrophy: Origin and correlation to clinical course. Muscle Nerve 18, 1115-1120 (1995).
48. Pavlath, G.K., Rich, K., Webster, S.G. & Blau, H.M. Localization of muscle gene products in nuclear domains. Nature 337, 570-573 (1989).
49. Ralston, E. & Hall, Z.W. Intracellular and surface distribution of a membrane protein (CD8) derived from a single nucleus in multinucleated myotubes. J. Cell Biol. 109, 2345-2352 (1989).
50. Ralston, E. & Hall, Z.W. Transfer of a protein encoded by a single nucleus to nearby nuclei in multinucleated myotubes. Science 244, 1066-1069 (1989).
51. Yoshida, M. & Ozawa, E. Glycoprotein complex anchoring dystrophin to sarcolemma. J. Biochem. 108, 748-752 (1990).
52. Ibraghimov-Beskrovnaya, O. et al. Primary structure of dystrophin-associated glyco-proteins linking dystrophin to the extracellular matrix. Nature 355, 696-702 (1992).
53. Roy, R. et al. Antibody formation after myoblast transplantation in Duchenne-dystrophic patients, donor HLA compatible. Transplant. Proc. 25, 995-997 (1993).
54. Pegoraro, E. et al. Genetic and biochemical normalization in female carriers of Duchenne muscular dystrophy: Evidence for failure of dystrophin production in dystrophin-competent myonuclei. Neurology 45, 677-690 (1995).
55. Webster, C., Pavlath, G.K., Parks, D.R., Walsh, F.S. & Blau, H.M. Isolation of human myoblasts with the fluorescence-activated cell sorter. Exp. Cell Res. 174, 252-265 (1988).
56. Lichter, P. et al. High-resolution mapping of human chromosome 11 by in situ hybridization with cosmid clones. Science 247, 64-69 (1989).
57. Landon, D.N. in Skeletal Muscle Pathology, (eds. Mastaglia, F.L. & Walton, S.J.) 1-87 (Churchill Livingstone, London, 1982).