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Novel Therapeutic Approach to the Neurodegenerative Disorder Huntington’s Disease (HD)

January 15, 2015

New findings from the lab of Dr. Beverly Davidson suggest groundwork for a novel therapeutic approach to the neurodegenerative disorder Huntington’s Disease (HD). In their experiments, study leader Beverly L. Davidson, PhD, director of the Center for Cellular and Molecular Therapeutics (CCMT), and co-investigators adjusted levels of mTORC1 in mice bred to model features of HD. They injected bioengineered viruses as a gene therapy tool to carry DNA that directed the production of regulatory proteins called Rheb and Rhes that act along the mTORC1 pathway. After the researchers restored mTORC1 activity to more normal levels, brain areas that had begun to atrophy recovered volume and permitted better motor function. The study was published in the journal Neuron and a substantial part of the research was conducted in Dr. Davidson’s laboratory at the University of Iowa before she joined the Department faculty as director of the CHOP CCMT in 2014.
Read the CHOP research announcement.

In other news, Dr. Davidson also recently joined the National Advisory Neurological Disorders and Stroke Council, the principal advisory body to the National Institute of Neurological Disorders and Stroke (NINDS).
Read the NINDS news release.