Dystrophin expression in the mdx mouse restored by stem cell transplantation

@article{Gussoni1999DystrophinEI,
  title={Dystrophin expression in the mdx mouse restored by stem cell transplantation},
  author={Emanuela Gussoni and Yuko Soneoka and Corinne D. Strickland and Elizabeth A. Buzney and Mohamed K Khan and A. P. F. Flint and Louis M. Kunkel and Richard C. Mulligan},
  journal={Nature},
  year={1999},
  volume={401},
  pages={390-394}
}
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… CONTINUE READING
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