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UVa researcher studying disease that cripples newborns

RNA and returned to normal. Only mice with severely damaged hearts did not recover.

We showed in our mouse model that when you make this poisonous RNA, the mice get various aspects of myotonic dystrophy, Dr. Mahadevan said. Then, when you take away the toxic RNA, the mice get back to normal.

The UVa study appeared in the September 2006 issue of Nature Genetics and can be found online at: http://www.nature.com/ng/index

Looking ahead, Dr. Mahadevan said, The goal of our work is to understand the molecular mechanisms underlying DM and to establish model systems for therapeutic interventions. Based on what weve learned so far, a therapy that silences the expression of the toxic RNA molecule seems to be a viable treatment for DM.

In the Rachel Fund announcement last month, Dr. Brenda Banwell with Muscular Dystrophy Canada (MDC) noted, the work of Dr. Mahadevans laboratory will provide novel insights into myotonic dystrophy and bridges bench research to clinical observation. We are pleased to fund excellence in research and encourage scientists working on the mechanisms and treatment of myotonic dystrophy to work together to further new insights into this disorder.


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Contact: Ellen C. McKenna
ecm6n@virginia.edu
434-982-4490
University of Virginia Health System
22-May-2007


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