[IE-01-2] Mechansistic and Therapeutic Insight into Expansion Repeat Disorders
Leonard Petrucelli, Ph.D., is a consultant and chair of the Department of Neuroscience at Mayo Clinic in Florida. Dr. Petrucelli and his research team are at the forefront of their field, researching the cellular mechanisms that cause neurodegeneration in Alzheimer’s disease, amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease, and frontotemporal dementia (FTD). By combining expertise in drug discovery, cell biology and induced pluripotent stem cell (iPSC) modeling, his lab aims to develop therapies for the treatment of diseases characterized by abnormal protein aggregation. Dr. Petrucelli’s team recently discovered a new therapeutic target and biomarker with the aim of improving the diagnosis and prognosis for patients suffering from FTD and ALS. His team’s research has been published in top tier journals including Science, Nature Medicine, Nature Neuroscience, Neuron, and Annals of Neurology.
Dr. Petrucelli is principal investigator for several grants funded by the National Institutes of Health (NIH) including R35 and is director of two funded NIH programs focused on c9orf72 and tau. He serves on the Scientific Advisory and is vice-chair to the Florida Alzheimer’s Disease Research Grant Advisory Board.
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