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 pre-clinical canine model of Duchenne muscular dystrophy (DMD). In recipients rendered tolerant to their dog leukocyte antigen (DLA)-matched unaffected littermates, through hematopoietic stem cell transplantation (HCT), intramuscular injection of donor marrow cells produced no evidence of dystrophin expression, and clonal analysis of satellite cells failed to reveal donor contribution.