Makani and Luzzatto, Of mice and men: From hematopoiesis in mouse models to curative gene therapy for sickle cell disease

Through studies in mice and in humans, Stuart Orkin showed that GATA-1 is a master transcriptional regulator of hematopoiesis. He has highlighted the role of BCL11A in the fetal-adult hemoglobin switch. The Gairdner Foundation Award recognizes Orkin’s contribution to the development of gene therapy of sickle cell disease.

View the rest of the article here

View the PDF