Mar. 5, 2013 Researchers at the University of Minnesota's Lillehei Heart Institute have combined genetic repair with cellular reprogramming to generate stem cells capable of muscle regeneration in a mouse model for Duchenne Muscular Dystrophy (DMD). The research, which provides proof-of-principle for the feasibility of combining induced pluripotent stem cell technology and genetic correction to treat muscular dystrophy, could present a major step forward in autologous cell-based therapies for DMD and similar conditions and should pave the way for testing the approach in reprogrammed human pluripotent cells from muscular dystrophy patients. The research is published in Nature Communications. To achieve a meaningful, effective muscular dystrophy therapy in the mouse model, University of Minnesota researchers combined three groundbreaking technologies. First, researchers reprogrammed skin cells into "pluripotent" cells -- cells capable of differentiation into any of the mature cell types within an organism. The researchers generated pluripotent cells from the skin of mice that carry mutations in the dystrophin and utrophin genes, causing the mice to develop a severe case of muscular dystrophy, much like the type seen in human DMD patients. This provided a platform that would mimic what would theoretically occur in human models. The second technology employed is a … Continue reading →

University of Minnesota researchers are one step closer to potential treatments for muscular dystrophy. According to an online study published Wednesday in the journal Nature Communications, researchers from the Universitys Lillehei Heart Institute have found a potential treatment for Duchenne muscular dystrophy in mice. The researchers combined cell therapy and genetic correction methods to turn the mices own cells into stem cells that can regenerate muscle. Its game-changing research, in my opinion, in the field of muscular dystrophy, said Jakub Tolar, director of the Universitys Stem Cell Institute. Muscular dystrophy is a genetic disorder that weakens and deteriorates muscles over time. Theres currently no effective treatment for it in humans, said Rita Perlingeiro, the studys principal investigator and a University professor. Perlingeiro said the research provides proof-of-principle for the possibility of treating the disorder and could translate to testing in human cells from dystrophic patients. Duchenne muscular dystrophy occurs mostly in children, who are often confined to wheelchairs as a result. Many children die from the disorder by the time theyre teenagers because the deterioration of the diaphragm prevents them from breathing, Perlingeiro said. The impact [of muscular dystrophy] on the individuals and their families is profound, lifelong and most … Continue reading →