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BCL11A is a gene encoding a regulatory protein that functions as a transcription factor. It plays critical roles in hematopoiesis, brain development, and fetal-to-adult hemoglobin switching. It is considered a proto-oncogene and is implicated in leukemia and lymphoma. Therapeutic targeting of BCL11A is under investigation for treating sickle cell disease and beta-thalassemia.
Targeting BCL11A to reactivate fetal hemoglobin
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