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| Dystrophin expression in the mdx mouse restored by stem cell transplantation |
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| Author(s): Gussoni E, Soneoka Y, Strickland CD, Buzney EA, Khan MK, Flint AF, Kunkel LM, Mulligan RC |
| Source: NATURE Volume: 401 Issue: 6751 Pages: 390-394 Published: SEP 23 1999 |
| Times Cited: 1,042 References: 29 |
| Abstract: The development of cell or gene therapies for diseases involving cells that are widely distributed throughout the body has been severely hampered by the inability to achieve the disseminated delivery of cells or genes to the affected tissues or organ(1). Here we report the results of bone marrow transplantation studies in the mdx mouse, an animal model of Duchenne's muscular dystrophy(2), which indicate that the intravenous injection of either normal haematopoietic stem cells or a novel population of muscle-derived stem cells into irradiated animals results in the reconstitution of the haematopoietic compartment of the transplanted recipients, the incorporation of donor-derived nuclei into muscle, and the partial restoration of dystrophin expression in the affected muscle. These results suggest that the transplantation of different stem cell populations, using the procedures of bone marrow transplantation, might provide an unanticipated avenue for treating muscular dystrophy as well as other diseases where the systemic delivery of therapeutic cells to sites throughout the body is critical. Our studies also suggest that the inherent developmental potential of stem cells isolated from diverse tissues or organs may be more similar than previously anticipated. |
| Document Type: Article |
| Language: English |
| Reprint Address: Mulligan, RC (reprint author), Childrens Hosp, Howard Hughes Med Inst, Boston, MA 02115 USA |
Addresses:
1. Childrens Hosp, Howard Hughes Med Inst, Boston, MA 02115 USA
2. Childrens Hosp, Div Genet, Boston, MA 02115 USA
3. Childrens Hosp, Dept Surg Res, Boston, MA 02115 USA
4. Harvard Univ, Sch Med, Dept Genet, Boston, MA 02115 USA |
| Publisher: MACMILLAN MAGAZINES LTD, PORTERS SOUTH, 4 CRINAN ST, LONDON N1 9XW, ENGLAND |
| Subject Category: Multidisciplinary Sciences |
| IDS Number: 240JJ |
| ISSN: 0028-0836 |
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