Abstract:
: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.
journal_name
Bloodjournal_title
Bloodauthors
Dell'Agnola C,Wang Z,Storb R,Tapscott SJ,Kuhr CS,Hauschka SD,Lee RS,Sale GE,Zellmer E,Gisburne S,Bogan J,Kornegay JN,Cooper BJ,Gooley TA,Little MTdoi
10.1182/blood-2004-06-2247subject
Has Abstractpub_date
2004-12-15 00:00:00pages
4311-8issue
13eissn
0006-4971issn
1528-0020pii
2004-06-2247journal_volume
104pub_type
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