Stem Cell Therapy Offers Hope to Spinal Muscular Atrophy Patients

Children born with spinal muscular atrophy (SMA) have a grim prognosis: most infants rarely see it to their second birthday. This debilitating, genetic disease has no cure; however, a study in Science and Translational Medicine demonstrates a possible stem cell therapy. Giacomo Comi and colleagues derived stem cell lines from skin samples of patients with SMA, and corrected a faulty genetic copy of the gene responsible for the disease. The researchers then reprogrammed these corrected cells into motor neurons. Transplanted in mice, the motor neurons improved disease phenotype and increased lifespan.

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