Tag: BCL11A
-
Nation & World
Gene therapy for sickle cell disease passes key preclinical test
A precision-engineered gene therapy virus, inserted into blood stem cells that are then transplanted, markedly reduced sickle-induced red-cell damage in mice with sickle cell disease, researchers from Dana-Farber/Boston Children’s Cancer and Blood Disorders Center reported in the Journal of Clinical Investigation.
-
Nation & World
Dialing down sickle cell disease
Flipping a single molecular switch can reverse illness in an animal model of sickle cell disease, according to a study by Harvard researchers at Children’s Hospital Boston and Dana-Farber Cancer Institute.