Of mice and men: From hematopoiesis in mouse models to curative gene therapy for sickle cell disease.
review · Level V
Where this comes from
- Record sourced from PubMed, PMID 35385685.
- Also identified by DOI 10.1016/j.cell.2022.03.031.
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Abstract
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.
Medical subject headings
- Anemia, Sickle Cell
- Awards and Prizes
- Genetic Therapy