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Nanoparticles treat muscular dystrophy in mice
Date:2/11/2014

ug itself can permeate the muscle tissue."

Compared with oral delivery, the nanoparticle approach also allowed the researchers to give the mice smaller doses of the drug.

"We showed that oral doses of rapamycin, even at 10 times the dose we used in the nanoparticles, were ineffective," Weihl said. "This is important because rapamycin suppresses the immune system, and directly targeting it to muscle in smaller doses would reduce unwanted side effects."

Current treatment for Duchenne involves corticosteroids such as prednisone, which has been shown to extend the time patients are able to walk. But steroids also cause weight gain, brittle bones, high blood pressure and other long-term side effects.

Although it's not clear why steroid treatment helps maintain skeletal muscle strength, Weihl said the study suggests prednisone also may promote autophagy, raising the possibility of combination therapy, in which both steroid treatment and rapamycin nanoparticles could be given simultaneously, each at lower doses.


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Contact: Julia Evangelou Strait
straitj@wustl.edu
314-286-0141
Washington University School of Medicine
Source:Eurekalert  

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