Dystrophin expression in the mdx mouse restored by stem cell transplantation.
Nature
; 401(6751): 390-4, 1999 Sep 23.
Article
em En
| MEDLINE
| ID: mdl-10517639
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. Here we report the results of bone marrow transplantation studies in the mdx mouse, an animal model of Duchenne's muscular dystrophy, 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.
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Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Distrofina
/
Transplante de Medula Óssea
/
Músculo Esquelético
/
Transplante de Células-Tronco Hematopoéticas
/
Distrofia Muscular Animal
Limite:
Animals
Idioma:
En
Revista:
Nature
Ano de publicação:
1999
Tipo de documento:
Article
País de afiliação:
Estados Unidos