HACKENSACK, N.J., March 21, 2013 /PRNewswire-USNewswire/ --Parent Project Muscular Dystrophy (PPMD) has awarded University of Minnesota researcher Dr. Rita Perlingeiro a grant for $220,000 to develop a therapeutic approach for Duchenne muscular dystrophy (Duchenne) using a new type of "adult-derived" stem cell called "induced pluripotent stem cells" or "iPSCs." iPSCs can be created from non-controversial sources like skin, have the ability to multiply indefinitely, and can be coaxed to become many different kinds of tissue types. (Logo: http://photos.prnewswire.com/prnh/20100119/DC39975LOGO) Previously, Dr. Perlingeiro's group demonstrated that skin cells from donor mice that lack dystrophin could be converted into iPSCs, and then converted again into muscle stem cells with the mutation corrected using a modified viral carrier. When these corrected mouse stem cells were transplanted into recipient mice that lacked dystrophin, the mice showed improved muscle strength. Now Dr. Perlingeiro will repeat this process with funding from PPMD, this time using human skin cells from donors with Duchenne and using a safer method of correcting the dystrophin deficiency that does not rely upon random integration. The goal is to start laying the groundwork for an FDA-approved stem cell treatment for Duchenne. "Translating results in mice to treatments in humans is never straightforward," said … Continue reading
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