Dystrophin expression in the mdx mouse restored by stem cell transplantation

Nature

Volumn 401, Issue 6751, 1999, Pages 390-394

  Gussoni, Emanuela ^a   Soneoka, Yuko ^a,b   Strickland, Corinne D ^a   Buzney, Elizabeth A ^a   Khan, Mohamed K ^a   Flint, Alan F ^a   Kunkel, Louis M ^a,b   Mulligan, Richard C ^a,b  

^a BOSTON CHILDREN S HOSPITAL   (United States)

^b HARVARD MEDICAL SCHOOL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

DYSTROPHIN;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CELL POPULATION; DUCHENNE MUSCULAR DYSTROPHY; HEMATOPOIESIS; MOUSE; MUSCLE; NONHUMAN; PRIORITY JOURNAL; PROTEIN EXPRESSION; STEM CELL TRANSPLANTATION;

ANIMALS; BONE MARROW TRANSPLANTATION; CELL DIFFERENTIATION; CELL NUCLEUS; CELL SEPARATION; DYSTROPHIN; FEMALE; GENE THERAPY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMATOPOIETIC STEM CELLS; IN SITU HYBRIDIZATION, FLUORESCENCE; MALE; MICE; MICE, INBRED C57BL; MUSCLE, SKELETAL; MUSCULAR DYSTROPHY, ANIMAL;

ANIMALIA;

EID: 0033598374     PISSN: 00280836     EISSN: None     Source Type: Journal    
DOI: 10.1038/43922     Document Type: Article

References (30)

1. Mulligan, R. C. The basic science of gene therapy. Science 260, 926-932 (1993).
2. Sicinski, P. et al. The molecular basis of muscular dystrophy in the mdx mouse: a point mutation. Science 244, 1578-1580 (1989).
3. Hall, Z. W. & Ralston, E. A. Nuclear domains in muscle cells. Cell 59, 771-772 (1989).
4. Karpati, G. et al. Dystrophin is expressed in mdx skeletal muscle fibers after normal myoblast implantation. Am. J. Pathol. 135, 27-32 (1989).
5. Pavlath, G. K., Rich, K., Webster, S. G. & Blau, H. M. Localization of muscle gene products in nuclear domains. Nature 337, 570-573 (1989).
6. Gussoni, E., Blau, H. M. & Kunkel, L. M. The fate of individual myoblasts after transplantation into muscles of DMD patients. Nature Med. 3, 970-977 (1997).
7. Hoffman, E. P., Morgan, J. E., Watkins, S. C. & Partridge, T. A. Somatic reversion/suppression of the mouse mdx phenotype in vivo. J. Neurol Sci. 99, 9-25 (1990).
8. Ferrari, G. et al. Muscle regeneration by bone marrow-derived myogenic progenitors. Science 279, 1528-1530 (1998).
9. Bittner, R. E. et al. Recruitment of bone marrow-derived cells by skeletal and cardiac muscle in adult dystrophic mdx mice. Anat. Embryol. (Berl.) 199, 391-396 (1999).
10. Goodell, M. A., Brose, K., Paradis, G., Conner, A. S. & Mulligan, R. C. Isolation and functional properties of murine hematopoietic stem cells that are replicating in vivo. J. Exp. Med. 183, 1797-1806 (1996).
11. Goodell, M. A. et al. Dye efflux studies suggest that hematopoietic stem cells expressing low or undetectable levels of CD34 antigen exist in multiple species. Nature Med. 3, 1337-1345 (1997).
12. 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).
13. Mauro, A. Satellite cells of skeletal muscle. J. Biophys. Biochem. Cytol. 9, 493-495 (1961).
14. Partridge, T. A., Morgan, J. E., Coulton, G. R., Hoffman, E. P. & Kunkel, L. M. Conversion of mdx myofibers from dystrophin negative to positive by injection of normal myoblasts. Nature 337, 176-179 (1989).
15. Partridge, T. A. Invited review: myoblast transfer: a possible therapy for inherited myopathies? Muscle Nerve 14, 197-212 (1991).
16. Acsadi, G. et al. Human dystrophin expression in mdx mice after intramuscular injections of DNA constructs. Nature 352, 815-818 (1991).
17. Acsadi, G. et al. Human dystrophin expression in mdx mice after intramuscular injections of DNA constructs. Nature 352, 815-818 (1991).
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. 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 b-galactosidase. Proc. Natl Acad. Sci. USA 93, 5731-5736 (1996).
20. Rafael, J. A. et al. Prevention of dystrophic pathology in mdx mice by a truncated dystrophin isoform. Hum. Mol. Genet. 3, 1725-1733 (1994).
21. 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).
22. Pereira, R. F. et al. Cultured adherent cells from marrow can serve as long-lasting precursor cells for bone, cartilage, and lung in irradiated mice. Proc. Natl Acad. Sci. USA 92, 4857-4861 (1995).
23. Saito, T. et al. Myogenic expression of mesenchymal stem cells within myotubes of mdx mice in vitro and in vivo. Tissue Eng. 1, 327-343 (1995).
24. Prockop, D. J. Marrow stromal cells as stem cells for nonhematopoietic tissues. Science 276, 71-74 (1997).
25. Asahara, T. et al. Isolation of putative progenitor endothelial cells for angiogenesis. Science 275, 964-967 (1997).
26. Shi, Q. et al. Evidence for circulating bone marrow-derived endothelial cells. Blood 92, 362-367 (1998).
27. Bjornson, C. R., Rietze, R. L., Reynolds, B. A., Magli, M. C. & Vescovi, A. L. Turning brain into blood: a hematopoietic fate adopted by adult neural stem cells in vivo. Science 283, 534-537 (1999).
28. Baroffio, A., Bochaton-Piallat, M.-L., Gabbiani, G. & Bader, C. R. Heterogeneity in the progeny of single human muscle satellite cells. Differentiation 59, 259-268 (1995).
29. Lichter, P. et al. Rapid detection of human chromosome 21 aberrations by in situ hybridization. Proc. Natl Acad. Sci. USA 85, 9664-9668 (1988).
30. Gussoni, E. et al. A method to codetect introduced genes and their products in gene therapy protocols. Nature Biotech. 14, 1012-1016 (1996).