A novel mouse model to screen compounds for the treatment of neurodegenerative disorders
Introduction Protein misfolding and aggregation are thought to play a central role in the pathogenesis of many, if not most, neurodegenerative disorders. The pharmaceutical industry has recently invested significant effort to identify small molecule inhibitors of protein misfolding and aggregation, which could be used to treat neurodegenerative disorders. However, one major obstacle is the lack of cost and time-effective in vivo models for tests of drug bioavailability, pharmacokinetics, pharmacodynamics and efficacy. Technology description Paul Muchowski’s laboratory has generated a novel mouse model to screen compounds for the treatment of neurodegenerative disorders. These transgenic mice express aggregationprone proteins that cause Huntington and Parkinson disease in their ocular lens. Expression of a mutant huntingtin fragment or α-synuclein in the lens leads to protein aggregation and cataract formation, which can be monitored in real time by noninvasive, highly sensitive optical techniques. These novel mouse models will facilitate the characterization of protein aggregation in vivo and can be used in efficient and economical screens for chemical and genetic modifiers of disease-relevant protein aggregation. Business Opportunity Approximately 40,000 Americans are diagnosed with Parkinson's disease each year, and this number does not reflect the thousands of cases that go undetected. Huntington's disease affects about 30,000 people in the United States alone, and at least 150,000 others have a 50 percent risk of developing Huntington's disease at some point. Intellectual Property Position There is a US patent pending on this technology: Non-Invasive Methods and Related Compositions for Identifying Compounds That Modify In Vivo Aggregations of Disease- Related Polypeptides, Appl. No. 10/918,708. Related Publication J. Biol. Chem., Vol. 283, Issue 10, 6330-6336, March 7, 2008 For more information on this technology contact:Valerie Carricaburu, Ph.D. Technology Manager, Invention Licensing [email protected], 205-543-3970
Type of Offer: Licensing
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