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Base editing shows potential superiority for curing sickle cell disease

July 3, 2023 sarah JonasResearch Results

Gene therapy that alters hemoglobin genes may be an answer to curing sickle cell disease and beta thalassemia. Scientists at St. Jude Children’s Research Hospital and the Broad Institute of MIT and Harvard found base editing increased fetal hemoglobin production in a new treatment.

CHOP Researchers Develop Proof-of-Concept Treatment that Elevates Both Adult and Fetal Hemoglobin

June 1, 2021 sarah JonasResearch Results

Researchers at Children’s Hospital of Philadelphia (CHOP) have developed a proof-of-concept treatment for blood disorders like sickle cell disease and beta-thalassemia that could raise hemoglobin levels by activating production of both fetal and adult hemoglobin. Using a viral vector engineered to reactivate fetal hemoglobin production, suppress mutant hemoglobin, and supply functional adult hemoglobin, the researchers developed an approach that could produce more hemoglobin through a single vector. The results were published in Haematologica.