Hematopoietic Cell Transplantation Directly into Dystrophic Muscle Fails to Reconstitute Satellite Cells and Myofibers

Chimerism analyses from peripheral blood, muscle biopsies, and cultured muscle satellite cells from a DMD-affected dog given intramuscular marrow injections from the unaffected HCT donor. Assays performed by PCR amplification of variable number tandem repeats [15] that differentiated donor and recipient. Muscle biopsies demonstrate contribution from both donor and recipient representative of the hematopoietic cells present in muscle tissue, whereas cultured satellite cells failed to show donor contribution (PB, peripheral blood).

Reverse-transcriptase-PCR reactions for dystrophin and β-actin in muscle biopsies from a DMD-affected dog demonstrate no dystrophin mRNA expression above the background after intramuscular marrow injections from the unaffected HCT donor.