By Elizabeth Dougherty – Dana-Farber Cancer Institute –
In the early 1970s, the field of genetics was confined to the study of bacteria and viruses. Conventional wisdom at the time was that the tools of molecular biology couldn’t be used to study human diseases. Human cells were just too hard to access, and too slow to reproduce, but also, the technology available was too crude. Some experts in the field thought that molecular biology would never be done in humans.
Stuart Orkin, MD, and a small number of other scientists were undeterred, and through their persistent efforts upended that skepticism. Orkin, a physician-scientist at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and the David G. Nathan Distinguished Professor of Pediatrics at Harvard Medical School, was chair of Dana-Farber’s Pediatric Oncology department from 2000-2016. By adopting and mastering new molecular and genetic tools as quickly as they appeared, he produced a litany of firsts: the first mapping of the genetics of a disease, thalassemia, an inherited blood disorder; the discovery of the first mechanisms that enable blood stem cells to specialize into functional red blood cells; the science behind the first approved CRISPR-based gene therapy; and more.
