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.
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