Possible New Target for Sickle Cell Disease and Beta Thalassemia Treatment Identified

Possible new target for sickle cell disease and beta thalassemia treatment identified

Harvard Medical School researchers at Boston Children's Hospital and Dana-Farber Cancer Institute have identified a new pathway that regulates the production of fetal hemoglobin, a form of hemoglobin that normally gets replaced soon after birth by adult hemoglobin.

New Target Discovered for Sickle Cell Disease, Beta Thalassemia

Researchers identified a biological pathway that could help produce healthy red blood cells in patients with inherited blood disorders like sickle cell disease.

Genome-Wide Analysis Identifies BACH2 as Potential Fetal Hemoglobin Activation Target

This pathway, which the authors dub the “BACH2-NRF2 axis,” is independent of the well-known BCL11A transcription regulator, the target of the approved cell therapy Casgevy for sickle cell disease (SCD). The post Genome-Wide Analysis Identifies BACH2 as Potential Fetal Hemoglobin Activation Target appeared first on GEN - Genetic Engineering and Biotechnology News.

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 …