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Ellen Sidransky, MD

Senior Investigator and Branch Chief, Medical Genetics Branch at National Human Genome Research Institute, National Institutes of Health
Location: Bethesda, MD United States

Dr. Ellen Sidransky is the branch chief of the Medical Genetics Branch and is a pediatrician and geneticist in the National Human Genome Research Institute at the National Institutes of Health (NIH). Dr. Sidransky received her MD from Tulane University and trained in pediatrics at Northwestern University, and in clinical genetics at the NIH. Her research interests includes both clinical and basic aspects of Gaucher and Parkinson’s diseases, studies of genetic modifiers and the correlation between genes and appearance, preclinical disease models and novel treatment strategies. She played a lead role in establishing the association between glucocerebrosidase and parkinsonism. Author of more than 280 publications, she continues to focus on the complexity found in "simple" Mendelian disorders, the role of lysosomal pathways in parkinsonism and the development of small molecule chaperones therapy. She is a 2024 recipient of the Breakthrough Prize in Life Sciences.


Associated Grants

  • Bidirectional Genome-wide CRISPRi/a Screening Using Ratiometric GCase Substrates to Identify Genetic Modulators of Lysosomal GCase Activity

    2026


  • IMPACT-PD: Implications of Polyamine and Glucosylceramide Transport in Parkinson’s Disease

    2024


  • Developing an Assay to Identify Glucocerebrosidase-enhancing Chaperones for the Treatment of Parkinson’s Disease

    2022


  • IMPACT-PD - Implications of Polyamine and Glucosylceramide Transport in Parkinson’s Disease

    2020


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