January 16, 2013
Gene And Cell Therapies Against Duchenne Muscular Dystrophy

A pair of reports demonstrate gene therapy and cell therapy benefits in animal models of the genetic muscle disease Duchenne Muscular Dystrophy (DMD). A gene therapy that introduces a subset of the dystrophin gene (which is defected in DMD) boosted muscle force in dogs.

"We placed the new microgene into a virus and then injected the virus into dystrophic dogs' muscles," Duan said. Following gene therapy, Duan's team examined the dogs for signs of muscle disease and measured muscle force in treated and untreated dogs. After careful evaluation of 22 dogs, Duan and colleagues found that the new version of micro-dystrophin not only reduced inflammation and fibrosis, it also effectively improved muscle strength.

Even if you do not know anyone with DMD the research into it still matters to you. Why? All of us suffer dystrophy (wasting away) of our muscles due to aging. This happens to us at a much slower rate than with with those who have genetic defects. But some potential therapies for DMD might also be useful for muscle rejuvenation.

Cell therapies against DMD hold the biggest hope of also offering benefit for old people whose muscles are shriveling. So a report from University of Illinois on a stem cell treatment for DMD also moves us closer to stem cell treatments for aged muscles.

CHAMPAIGN, lll. Researchers have shown that transplanting stem cells derived from normal mouse blood vessels into the hearts of mice that model the pathology associated with Duchenne muscular dystrophy (DMD) prevents the decrease in heart function associated with DMD.

The stem cells helped prevent heart muscle deterioration.

In the new study, the researchers injected stem cells known as aorta-derived mesoangioblasts (ADM) into the hearts of dystrophin-deficient mice that serve as a model for human DMD. The ADM stem cells have a working copy of the dystrophin gene.

This stem cell therapy prevented or delayed heart problems in mice that did not already show signs of the functional or structural defects typical of Duchenne muscular dystrophy, the researchers report.

Support muscular dystrophy research. Among the hearts it will save could be your own.

Share |      Randall Parker, 2013 January 16 10:38 PM 


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