Professor Stuart Orkin
King Faisal Prize in Medicine 2020 Laureate
Topic: "Haemoglobinopathies"
After years of study, we have a deep understanding of how to apply gene therapy to provide effective and long-lasting benefits to patients with hemoglobinopathies.
Stuart Orkin received his B.S. degree from MIT and MD degree from Harvard Medical School (HMS) in 1972. He trained in general pediatrics and hematology/oncology at Boston Children’s Hospital, and served as a postdoctoral Research Associate at the NIH in the Laboratory of Philip Leder in 1973-1975. Following his appointment to the HMS faculty in 1978, he ascended to the rank of Professor in 1986. He served as the Chairman of the Department of Pediatric Oncology at Dana Farber Cancer Institute from 2000-2016. Currently, Professor Orkin is the David G. Nathan Distinguished Professor of Pediatrics at HMS, and an Investigator of the Howard Hughes Medical Institute.
Professor Orkin’s research has been on the forefront of molecular blood cell development and genetics for nearly 4 decades. His early work led to the first comprehensive molecular dissection of an inherited disorder (the thalassemia syndromes). He characterized genes responsible for other human blood disorders, including X-linked chronic granulomatous disease (the first positional cloning of a disease gene). Orkin identified the first hematopoietic transcription factors (the GATA family) and characterized their roles in blood cell development and cancer. His recent studies on BCL11A, a critical repressor of fetal hemoglobin (HbF), have illuminated regulation of the fetal-to-adult switch and improved prospects for HbF reactivation as genetic or pharmacological therapy of the thalassemias and sickle cell disease.
An author of 600 papers, Professor Orkin is an elected member of the National Academy of Sciences (NAS), National Academy of Medicine (NAM), American Academy of Arts and Sciences, and American Philosophical Society. He has been recognized with numerous awards, including the E. Mead Johnson Award of the American Academy of Pediatrics, Warren Alpert Prize, Helmut Horten Foundation Prize, Distinguished Research Award of the Association of American Medical Colleges, E. Donnall Thomas, Dameshek and Basic Science Mentor Awards of the American Society of Hematology, Jessie Stevenson Kovalenko Medal of the NAS, William A. Allan Award of the American Society of Human Genetics, George M. Kober Medal of the American Association of Physicians, and the Mechthild Esser Nemmers Prize in Medical Science.
This biography was written in the year the prize was awarded.
