Wednesday, September 7, 2016
Doris Duke Supports Research on Gene Therapy for Sickle Cell Disease
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 report today in the Journal of Clinical Investigation.
The work sets the stage for bringing a decades-old discovery about sickle-cell disease to the bedside.
A clinical gene therapy trial, using a virus rendered harmless in the laboratory, is expected to launch in the coming year...