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News for Healthier Living

Dana-Farber Research Reveals Possible New Target for Treatment of Sickle Cell and Beta Thalassemia

Gene therapy for blood disorders such as sickle cell disease and beta thalassemia switch on fetal hemoglobin and replace faulty adult hemoglobin by regulating levels of BCL11A, which was identified by Dana-Farber research two decades ago. In this new research, Dana-Farber investigators - including Vijay Sankaran, MD, PhD, who was involved in the discovery of the role BCL11A in blood disorders - have identified a new pathway that regulates fetal hemoglobin. That pathway involves BACH2 and NRF2 and was discovered using a large genome-wide association study that enabled an analysis of genes related to fetal hemoglobin from a wide range of ancestries, including European, African, and Asian ancestries. According to the study, BACH2 normally acts as a brake on fetal hemoglobin production. Reducing BACH2 enables NRF2 to activate fetal hemoglobin genes. The study also found that pharmacological inhibition of BACH2 increases fetal hemoglobin in human red blood cell precursors.

September 30, 2026


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