Muscular dystrophy cell therapy

The muscular dystrophies are characterized by progressive weakness and wasting of striated muscle and are caused by inherited or de novo gene mutation of sarcolemma-associated proteins or nuclear membrane–associated proteins. Patients suffering from muscular dystrophies can obtain a precise diagnosis of their underlying molecular defect, but no efficient treatment to prevent disability and death has been available. In the July 10 Sciencexpress, Maurilio Sampaolesi and colleagues from the Stem Cell Research Institute at San Raffaele Hospital show that that intraarterial delivery of mesoangioblasts can correct the molecular defect in a murine model of muscular dystrophy (Sciencexpress, DOI:10.1126/science.1082254, July 10, 2003).

Sampaolesi et al. injected [14C]thymidine-labeled wildtype mesoangioblasts into the right femoral artery of 1-month-old α-sarcoglycan (α-SG) null mice—a model for limb-girdle muscular dystrophy. They observed that this form of cell delivery was effective in restoring expression of α-SG protein and of the other members of the dystrophin-glycoprotein complex in the ...