sickle cell disease

Harvard researchers find new gene pathway for sickle cell treatment

A team of researchers at Boston Children's Hospital, Dana-Farber Cancer Institute, and the Broad Institute has discovered a previously unknown genetic pathway that activates fetal hemoglobin, opening a potential new front in the treatment of sickle cell disease and beta…

Gene-editing therapy hits 100% remission across 30-plus sickle cell and thalassemia patients

A base-editing therapy developed by Shanghai-based CorrectSequence Therapeutics has achieved transfusion independence or freedom from painful vaso-occlusive crises in every patient treated to date — more than 30 across four continents — according to clinical data published September 7 in…

CRISPR therapy shows promise in children as young as 5 with sickle cell disease

HCA Healthcare announced on Monday that research published in The New England Journal of Medicine demonstrates promising results from a CRISPR gene-editing therapy tested in children ages 5 to 11 with severe sickle cell disease and transfusion-dependent beta thalassemia, marking…