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U of M researchers utilize genetically corrected stem cells to spark muscle regeneration

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.

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Providing hope for people with rare diseases

February 28 is observed internationally as Rare Disease Day. Nearly 30 million people in the United States suffer from conditions classified as “rare diseases”.

While some bear names you’ve likely never heard of, other examples may surprise you …

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The University of Minnesota is a powerhouse in muscular dystrophy research

University of Minnesota faculty received 3 national grants awarded by the Muscular Dystrophy Association (MDA) on August 1, 2012.

The recipient faculty members are:

  • Dr. Atsushi Asakura, assistant professor of Neurology, investigating how blood vessels affect muscle stem cells and regeneration in muscular dystrophy.
  • Dr. Joseph Metzger, professor and chair of integrative biology and physiology, developing membrane sealants as therapeutics for Duchenne and Becker muscular dystrophies.
  • Dr. Rita Perlingeiro, associate professor of Medicine, investigating genetic correction and muscle regeneration in Duchenne and Becker muscular dystrophies.
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