Current Research
Hematopoietic Stem Cell Biology: Development of Blood Cell Lineages and Oncogenesis

Stuart Orkin is interested in how commitment to specific blood lineages is programmed and how cell-specific patterns of gene expression are established. Orkin and his team focus on the transcription factors crucial for development of stem cells or individual lineages, how those factors are regulated, and how aberration in those processes can lead to malignancy. Using human genetics and gene editing, they elucidated how the developmental switch from fetal to adult hemoglobin is controlled – work leading to clinical trials for reactivation of fetal hemoglobin as a therapy for sickle cell disease and beta-thalassemia. Building on this, the team seeks to use chemical approaches to mimic genetic strategies for fetal hemoglobin reactivation.

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