Stuart Holland Orkin is an American physician, stem cell biologist and researcher in pediatric hematology-oncology.
He did postdoctoral research in molecular biology at the National Institutes of Health under geneticist Philip Leder.
While Orkin was completing his training in hematology-oncology, his department chair, David G. Nathan, allowed him to establish his own research laboratory.
[4][5] In the 1970s and 1980s, Orkin conducted research that identified genetic mutations associated with a group of blood disorders known as the thalassemias.
His group demonstrated that loss of BCL11A alone is sufficient to rescue the phenotype of sickle cell disease (SCD).
In September 2015, Orkin published a study in the journal Nature showing a small section of DNA which could be responsive to gene therapy for sickle-cell disease.
[9] Elected to the National Academy of Sciences in 1991, Orkin won the Jessie Stevenson Kovalenko Medal from that organization in 2013.