Journal
BLOOD
Volume 104, Issue 13, Pages 4311-4318Publisher
AMER SOC HEMATOLOGY
DOI: 10.1182/blood-2004-06-2247
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Funding
- NCI NIH HHS [CA78902, P01 CA078902, CA15704] Funding Source: Medline
- NIAMS NIH HHS [AR18860] Funding Source: Medline
- NICHD NIH HHS [HD47175] Funding Source: Medline
- NIDDK NIH HHS [DK56465] Funding Source: Medline
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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 DIMID, 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. (C) 2004 by The American Society of Hematology.
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