University researchers find potential MD treatment option

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 commonly tragic, Tolar said. So anything that can be done to reverse this progressive disability is of enormous clinical impact.

Perlingeiro has worked on muscular dystrophy research for a decade. She oversaw the first successful use of human stem cells in treating the disease last year.

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University researchers find potential MD treatment option