STUART ORKIN received Boston Children’s Hospital’s Lifetime Impact Award at the hospital’s third annual Global Pediatric Innovation Summit.
Orkin is associate chief of hematology/oncology at Boston Children’s Hospital and chair of pediatric oncology at Dana-Farber Cancer Institute.
“Dr. Orkin’s contributions to the patients, families and staff from both our hospitals have been immeasurable,” said Boston Children’s Hospital President and CEO Sandra Fenwick. “For all of his dedication to research and care, he has never lost sight of teaching the next generation of researchers and caregivers, and we have all learned so much from him, particularly when it comes to commitment to excellence.”
Orkin’s laboratory was one of the first to apply molecular biology and DNA sequencing techniques to thalassemia, a blood disorder characterized by defects in genes that provide the instructions for producing hemoglobin. In addition, he has systematically dissected the hematopoietic process, identifying nearly every one of the master genes called transcription factors that regulate the development of every cell type found in the blood.
“Stu has always been in the vanguard when it comes to expanding our understanding of gene regulation, hematopoiesis and how they can go awry to cause blood disorders and leukemias,” said David Williams, president of Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. “We will continue to see the impact of his work as a scientist, a leader and a mentor for years to come.”
In recent years, his laboratory has studied the roles of two molecular switches—gene BCL11A and an enhancer that controls its activity—in controlling production of the adult and fetal forms of hemoglobin. Sickle cell anemia and thalassemia are both caused by mutations in adult hemoglobin. Orkin and his collaborators are attempting to use gene editing technologies such as CRISPR to manipulate BCL11A’s enhancer and force red blood cells to dial down adult hemoglobin production in favor of the fetal form.
Orkin also is a member of the National Academy of Sciences, the National Academy of Medicine and the American Academy of Arts and Sciences. He is a fellow of the American Association for the Advancement of Science, an investigator with the Howard Hughes Medical Institute, and the David G. Nathan Professor of Pediatrics at Harvard Medical School.
