Open AccessOf mice and men: From hematopoiesis in mouse models to curative gene therapy for sickle cell disease
Author(s) -
Julie Makani,
Lucio Luzzatto
Publication year - 2022
Publication title -
cell
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 26.304
H-Index - 776
eISSN - 1097-4172
pISSN - 0092-8674
DOI - 10.1016/j.cell.2022.03.031
Subject(s) - biology , genetic enhancement , haematopoiesis , disease , fetal hemoglobin , regulator , gene , cell , immunology , fetus , genetics , stem cell , medicine , pregnancy
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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