Dr. Stuart Orkin is a preeminent molecular biologist and leading authority in the field of blood cell development and genetic blood disorders. He currently serves as the David G. Nathan Distinguished Professor of Pediatrics at Harvard Medical School and is an Investigator at the Howard Hughes Medical Institute based at Boston Children's Hospital. After earning his BS from MIT in 1967 and his MD from Harvard Medical School in 1972, he conducted postdoctoral research at the National Institutes of Health under geneticist Philip Leder. Dr. Orkin joined the Harvard Medical School faculty in 1978, rising to full professor in 1986, and served as Chairman of the Department of Pediatric Oncology at the Dana-Farber Cancer Institute from 2000 to 2016.
Dr. Orkin's groundbreaking research has defined the molecular basis of numerous human blood disorders and illuminated the mechanisms governing blood cell development. His pioneering work provided the first comprehensive molecular dissection of the thalassemia syndromes, characterized genes responsible for other blood disorders including X-linked chronic granulomatous disease through the first positional cloning, and identified the first hematopoietic transcription factors of the GATA family. His seminal studies of BCL11A as a repressor of fetal hemoglobin have fundamentally advanced understanding of globin gene switching and created new therapeutic pathways for reactivating fetal hemoglobin as treatment for thalassemias and sickle cell disease. This work has transformed the diagnosis and treatment approaches for these debilitating genetic conditions, offering hope for millions of patients worldwide.
As an elected member of the National Academy of Sciences, National Academy of Medicine, American Academy of Arts and Sciences, and American Philosophical Society, Dr. Orkin has profoundly shaped the field of hematology and molecular medicine. His laboratory continues to investigate how commitment to specific blood lineages is programmed and how cell-specific gene expression patterns are established, with research encompassing molecular biology and contemporary mouse genetics. Dr. Orkin has received numerous prestigious honors including the Kovalenko Medal from the National Academy of Sciences and the King Faisal Prize in Medicine, recognizing his transformative contributions to medical science. Through his continued research, mentorship of future scientists, and leadership in the field, Dr. Orkin remains at the forefront of advancing our understanding of blood disorders and developing innovative therapeutic approaches.