2020 - Stuart Orkin

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.

Nationality: United States of America
Born: 1946, New York, United States of America
Professor Stuart Orkin was awarded the prize for his outstanding contributions to the field of hemoglobinopathies, which included seminal contributions of basic discoveries as well as translating them into clinical care in at least 3 areas. The first is the identification of mutations causing beta thalassemia syndromes; the second is studies of GATA-1 defining its function in red blood cell formation; and the third is the identification of BCL11A as the suppressor of hemoglobin F and understanding its mechanism of action. These works have led to clinical trials on the effect of this drug that improves beta-thalassemia and sickle cell status by increasing the level of F-pigment
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