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Blood, 15 December 2004, Vol. 104, No. 13, pp. 4311-4318. Prepublished online as a Blood First Edition Paper on August 24, 2004; DOI 10.1182/blood-2004-06-2247. This Article Full Text Full Text (PDF) All Versions of this Article: 2004-06-2247v1 104/13/4311    most recent Alert me when this article is cited Alert me if a correction is posted Citation Map Services Email this article to a friend Similar articles in this journal Similar articles in PubMed Alert me to new issues of the journal Download to citation manager Rights and Permissions Citing Articles Citing Articles via HighWire Citing Articles via CrossRef Citing Articles via Google Scholar Google Scholar Articles by Dell'Agnola, C. Articles by Little, M.-T. Search for Related Content PubMed PubMed Citation Articles by Dell'Agnola, C. Articles by Little, M.-T. Related Collections Hematopoiesis and Stem Cells Transplantation Gene Expression Related Article in Blood Online Social Bookmarking                   What's this?  Previous Article  |  Table of Contents TRANSPLANTATION Hematopoietic stem cell transplantation does not restore dystrophin expression in Duchenne muscular dystrophy dogs Chiara Dell'Agnola, Zejing Wang, Rainer Storb, Stephen J. Tapscott, Christian S. Kuhr, Stephen D. Hauschka, Richard S. Lee, George E. Sale, Eustacia Zellmer, Serina Gisburne, Janet Bogan, Joe N. Kornegay, Barry J. Cooper, Theodore A. Gooley, and Marie-Térèse Little From the Fred Hutchinson Cancer Research Center, Seattle, WA; the University of Washington, Seattle, WA; the University of Missouri, Columbia, MO; and Oregon State University, Corvallis, OR. Duchenne muscular dystrophy (DMD) is caused by mutations in the dystrophin gene on the X-chromosome that result in skeletal and cardiac muscle damage and premature death. Studies in mice, including the mdx mouse model of DMD, have demonstrated that circulating bone marrow–derived cells can participate in skeletal muscle regeneration, but the potential clinical utility of treating human DMD by allogeneic marrow transplantation from a healthy donor remains unknown. To assess whether allogeneic hematopoietic cell transplantation (HCT) provides clinically relevant levels of donor muscle cell contribution in dogs with canine X-linked muscular dystrophy (c-xmd), 7 xmd dogs were given hematopoietic cell (HC) transplants from nonaffected littermates. Compared with the pretransplantation baseline, the number of dystrophin-positive fibers and the amount of wild-type dystrophin RNA did not increase after HCT, with observation periods ranging from 28 to 417 days. Similar results were obtained when the recipient dogs were given granulocyte colony-stimulating factor (G-CSF) after their initial transplantation to mobilize the cells. Despite successful allogeneic HCT and a permissive environment for donor muscle engraftment, there was no detectable contribution of bone marrow–derived cells to either skeletal muscle or muscle precursor cells assayed by clonal analyses at a level of sensitivity that should detect as little as 0.1% donor contribution. CiteULike    Connotea    Del.icio.us    Digg    Reddit    Technorati    What's this? Related Article in Blood Online: HSCs for muscular dystrophy: a paradigm shift, back John F. Tisdale Blood 2004 104: 3847-3848. [Full Text] [PDF] This article has been cited by other articles: S. Li, E. Kimura, R. Ng, B. M. Fall, L. Meuse, M. Reyes, J. A. Faulkner, and J. S. Chamberlain A highly functional mini-dystrophin/GFP fusion gene for cell and gene therapy studies of Duchenne muscular dystrophy Hum. Mol. Genet., May 15, 2006; 15(10): 1610 - 1622. [Abstract] [Full Text] [PDF] G. Wernig, V. Janzen, R. Schafer, M. Zweyer, U. Knauf, O. Hoegemeier, R. R. Mundegar, S. Garbe, S. Stier, T. Franz, et al. The vast majority of bone-marrow-derived cells integrated into mdx muscle fibers are silent despite long-term engraftment PNAS, August 16, 2005; 102(33): 11852 - 11857. [Abstract] [Full Text] [PDF]

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