autologous myogenic cell therapy

Autologous myogenic cell therapy is an experimental cell-based therapeutic approach being developed by Radboud University Medical Center to treat the neuromuscular phenotype of myotonic dystrophy type 1 (DM1). The therapy involves isolating autologous pericytes—multipotent, vessel-associated progenitor cells—from a patient's skeletal muscle biopsy. These cells may be genetically corrected ex vivo using CRISPR/Cas9 to remove the pathogenic CTG-repeat expansion in the DMPK gene. Once systemically or locally administered, these myogenic cells are intended to renew the muscle progenitor pool and fuse with existing myotubes to form regenerating fibers, thereby addressing muscle wasting and weakness.

Other names

autologous pericyte therapyautologous myogenic progenitor cell therapy