Hematopoietic cell transplantation directly into dystrophic muscle fails to reconstitute satellite cells and myofibers.
We sought to determine whether wild-type hematopoietic cell transplantation directly into muscle could restore dystrophin expression in a relevant preclinical canine model of Duchenne muscular dystrophy. In recipients rendered tolerant to their dog leukocyte antigen-matched unaffected littermates through hematopoietic stem cell transplantation, intramuscular injection of donor marrow cells produced no evidence of dystrophin expression, and clonal analysis of satellite cells failed to reveal any donor contribution.
Kuhr
Christian S
CS
Clinical Research Division, Fred Hutchinson Cancer Research Center, and Department of Surgery, University of Washington School of Medicine, Seattle, Washington 98101, USA. christian.kuhr@vmmc.org
Lupu
Marilena
M
Storb
Rainer
R
D000818:Animals; D002454:Cell Differentiation / Q000502:physiology; D004195:Disease Models, Animal; D004285:Dogs; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006412:Hematopoietic Stem Cells / Q000502:physiology*; D007273:Injections, Intramuscular; D009132:Muscles / Q000166:cytology*; D020388:Muscular Dystrophy, Duchenne / Q000628:therapy*; D009210:Myofibrils; D032496:Satellite Cells, Skeletal Muscle; D017211:Treatment Failure