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UMMS and Lundbeck to explore potential targeted therapy for Huntington's disease
Date:1/28/2011

WORCESTER, Mass The University of Massachusetts Medical School (UMMS) and Lundbeck Inc. today announced a research collaboration aimed at further development of a targeted therapy to slow or halt the progression of Huntington's disease (HD).

At this time, there is no way to stop or reverse the course of HD, a challenging hereditary neurodegenerative disease characterized by a triad of progressive motor, cognitive and emotional symptoms. This collaboration will support a distinguished group of scientists in the study of RNAi-based therapies as a possible method for selectively suppressing production of mutant huntingtin (mHtt), the abnormal protein that causes HD.

RNA interference, or RNAi, is a natural process that cells use to turn down, or silence, activity of specific genes. HD is mapped to a specific gene, which makes it a promising target for RNAi-based therapy because production of a mutant protein like mHtt can potentially be blocked by knocking down, or reducing, the gene's activity.

"Our core idea is that RNAi can be used to selectively reduce mutant huntingtin production to slow or block the progression of HD, but we also hypothesize that excessive huntingtin silencing may impair neuronal function by interfering with essential signaling events," said Neil Aronin, MD, professor in medicine and cell biology at UMMS and principal investigator of the study. "This research collaboration allows us to test promising RNAi-based therapeutic vehicles to selectively knock down mutant huntingtin with the goal of restoring normal neuronal function. We've come a long way in pushing this research forward, and this next step with Lundbeck is extremely exciting."

RNAi technology is used to interfere with the expression of a specific gene and, in this case, researchers will apply it to mHtt. This multi-faceted pre-clinical study progresses beyond previously successful mouse studies and could bring RNAi-based therapy one step closer
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Contact: Alison Duffy
alison.duffy@umassmed.edu
508-856-2000
University of Massachusetts Medical School
Source:Eurekalert

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