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The Neurodegenerative Diseases Laboratory of Leonard Petrucelli, Ph.D., is at the forefront of research investigating the cellular mechanisms that cause diseases characterized by abnormal protein aggregation such as Alzheimer's disease, frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). In expanding upon his commitment to understanding the causes of such diseases, Dr. Petrucelli is now emphasizing translational research geared toward identifying and developing therapies for treatment and prevention.
Dr. Petrucelli's lab aims to identify molecular culprits, including identifying the role epigenetic events may play in disease, and to develop therapeutic strategies to improve the prognosis for patients with neurodegenerative disorders. By combining expertise in drug discovery, cell biology and induced pluripotent stem cell (iPSC) modeling, his lab aims to design and optimize selective and potent compounds that can be developed into a therapy for patients who have C9orf72-related disorders and develop therapeutic inhibitors for the treatment of Alzheimer's disease and FTD. Given no biomarker currently exists for clinicians to definitively diagnose ALS or FTD, identifying potential biomarkers and therapeutic targets may greatly aid future drug discovery.
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