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CCMT Seminar - Mar 21, 2023
3500 Civic Center Blvd
Philadelphia, PA 19104
The Raymond G. Perelman Center for Cellular and Molecular Therapeutics at CHOP presents a seminar series:
Insights into Pathogenic Mechanisms in Huntington's Disease
Leslie M. Thompson, PhD
Donald Bren and Chancellor's Professor
Departments of Psychiatry and Human Behavior and Neurology and Behavior
University of California, Irvine
Description of Research
Dr. Thompson has studied Huntington’s disease (HD) for most of her scientific career and was a member of the international consortium that identified the causative gene for HD in 1993. She also co-identified the mutation causing achondroplasia, the most common genetic form of short-limbed dwarfism, in 1994. Since that time, the Thompson laboratory has been actively engaged in investigating the fundamental molecular and cellular events that underlie how the mutant HD gene causes degeneration of specific brain cell populations to induce motor and cognitive decline and premature death of patients with the ultimate goal to develop new treatments, including stem-cell based treatments. The laboratory also focuses on understanding mechanisms that underlie amyotrophic lateral sclerosis (ALS) and more recently X-linked Dystonia-Parkinsonism with the goal of developing personalized treatment strategies for these diseases. The research benefits from the integrated use of patient stem cells and mouse models of disease together with the studies of RNA biology, protein quality control and network-based bioinformatics. Dr. Thompson is a member of the Packard Center SAB, chair of the Huntington’s Disease Society of America SAB, chair-elect of the Hereditary Disease Foundation SAB, and is founding co-editor in chief of the Journal of Huntington’s Disease. She is the PI of the OMICS core of the Answer ALS program, which is a precision medicine approach to understand sporadic ALS in over 1000 ALS subjects and is a co-director of the Institute for Precision Health at UCI that seeks to improve health and advance treatments through AI and data driven technologies.
Host: Beverly Davdison, PhD
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