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

to human clinical trials. The study will evaluate potential dosing regimens of siRNA (small interfering RNA), packaged as short hairpin RNA (shRNA) and transported via the adeno-associated virus (AAV), a promising therapeutic vehicle for siRNA delivery to neurons. Additionally, researchers will work to establish the best brain distribution pattern for potential use of siRNA in clinical HD therapies. More specifically, a major element of the study will be to measure the volume of distribution in brain tissue of AAV-delivered shRNAmir (micro RNA-adapted shRNA).This will permit researchers to evaluate the dosing of AAV-shRNAmir in order to achieve spread throughout the striatum and nearby cortex.

"We've followed Dr. Aronin and his team of researchers at the Medical School for some time and have been inspired by their bold exploration of RNAi technology and its potential use as a therapy for HD," said Stevin Zorn, PhD, executive vice president, Lundbeck Research USA. "Lundbeck is proud to collaborate with such an exceptional group of scientists who are so devoted to those affected by this debilitating condition."

In addition to Dr. Aronin, a talented group of scientists comprise the UMMS research team, including: Guangping Gao, PhD, who brings vast experience in developing adeno-associated virus; Richard Moser, MD, a neurosurgeon with expertise in resection of brain tumors and accessing brain compartments; and Marian DiFiglia, PhD, of Massachusetts General Hospital, an expert in the neuropathology and mechanisms of HD. Consultants on the project include: Kitty Clarence-Smith, MD, PhD, Lundbeck Inc., a neuro-pharmacologist who was instrumental in the approval process of the first FDA-approved drug for HD chorea; Robert Friedlander, MD, Professor and Chair of Neurosurgery, University of Pittsburgh School of Medicine, who has expertise in the study of neuronal survival in HD; and Michael Levine, MD, Professor and Chair of the Neuro-Psychiatric Institu
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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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