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Scientists discover 'needle in a haystack' for muscular dystrophy patients
Date:1/22/2013

potentially important translational implications for therapy of Duchenne muscular dystrophy. The data from the Duan laboratory, reported in this paper and previous studies, demonstrates that the structural elements required for proper nNOS localization should be included in any DMD therapy for which dystrophin restoration is the goal."

For more than 10 years, Duan has been a leader in muscular dystrophy, gene therapy and biology research at MU. In addition to his recently published study in PNAS, Duan's lab continues to examine the basic scientific mechanisms behind muscular dystrophy as well as strategies for treating the disease. For example, his lab is studying the effectiveness of a gene therapy for treating heart failure associated with Duchenne muscular dystrophy.

Using viruses as a means for delivering gene therapy, Duan is also testing how synthetic microgenes could improve muscle function in dystrophic dog and mouse models. In 2011, he and Lai were granted a patent for a synthetic microgene developed in his lab that has now proved to enhance muscle function in dogs. Those results were also published this month in the journal Molecular Therapy.


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Contact: Laura Gerding
gerdingla@health.missouri.edu
573-882-9193
University of Missouri School of Medicine
Source:Eurekalert  

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Scientists discover 'needle in a haystack' for muscular dystrophy patients
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