Novel compounds and novel protein targets for the treatment of Parkinson's Disease

MEDI 285

Paul J. Hergenrother,, Department of Chemistry, University of Illinois, 600 S. Mathews Box 36-5, Urbana, IL 61801
As the ability to diagnosis neurodegenerative disorders at an early stage increases, neuroprotective therapies become more and more attractive. However, few protein targets have been validated for the treatment of these disorders. This lecture will detail recent findings indicating that inhibitors of the enzyme poly(ADP-ribose) glycohydrolase (PARG) have potential against Parkinson's disease. The discovery of a potent, cell permeable, small molecule inhibitor of PARG will be described, as will detailed structure-activity-relationship data. The efficacy of this inhibitor in both cell culture and mouse models of Parkinson's disease will be discussed.