autologous myogenic stem cells

Autologous myogenic stem cells are an investigational cell therapy developed by Academisch Ziekenhuis Maastricht (Maastricht University Medical Center) for the treatment of mitochondrial myopathy, specifically in patients with the m.3243A>G mutation. The therapy involves the isolation of satellite cells (myogenic precursors) from a patient's skeletal muscle biopsy. In patients with this specific mitochondrial DNA (mtDNA) mutation, satellite cells often exhibit significantly lower levels of mutation heteroplasmy compared to mature muscle fibers. These cells are expanded ex vivo and subsequently re-injected into the patient's muscle. The intended mechanism is for these low-heteroplasmy cells to fuse with existing muscle fibers or form new ones, thereby shifting the overall heteroplasmy toward healthy mtDNA and restoring mitochondrial oxidative phosphorylation capacity.

Other names

autologous satellite cellsautologous myoblastsautologous satellite cell-derived myoblasts