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Dr. Ellen Sidransky

Editor (Genetics)

Dr. Ellen Sidransky is Chief of the Section of Molecular Neurogenetics and is a pediatrician and clinical geneticist in the Medical Genetics Branch of the National Human Genome Research Institute at the National Institutes of Health in Bethesda, Maryland. Dr. Sidransky received her B.A. in Biology from Brandeis University and her M.D. from Tulane University. She then trained in pediatrics at Children's Memorial Hospital/Northwestern University, and received her fellowship training in clinical genetics at the NIH Genetics Training Program. Dr. Sidransky joined the NIH and began studying Gaucher disease in the laboratory of Dr. Edward Ginns, and has been a tenured investigator and Section Chief since 2000.

Dr. Sidransky's research includes both clinical and basic research aspects of Gaucher disease and Parkinson disease, studies of genotype/phenotype correlation and genetic modifiers, clinical insights from mouse models, and the development of new treatment strategies for the lysosomal storage disorders. She has played a lead role in establishing the association between glucocerebrosidase and Parkinson disease, including spearheading an international collaboration published in the New England Journal of Medicine. Her current research also focuses on understanding the complexity encountered in "simple" Mendelian disorders and the development of small molecule chaperones as a therapy for Gaucher disease and related disorders.  The author of over 200 publications, Dr. Sidransky directs NIH clinical protocols, evaluating patients with lysosomal storage disorders and prospectively studying patients with Parkinson disease harboring GBA1 mutations and their relatives. In this editorial role she is serving in her personal capacity, and that any views expressed are not representative of the NIH or the US Federal government. 


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The Rare Haematology Resource Centre is funded by Sanofi Genzyme and developed by Elsevier. Sanofi Genzyme has no editorial control over the content of this Resource Centre. The Resource Centre and all content therein has been subject to an independent editorial review. The content expressed are those of the individual experts and the editorial board governed by Elsevier and do not necessarily express the view of Sanofi Genzyme.