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.
The new gene therapy is the culmination of research going back to the 1980s that found that sickle cell disease is milder in people whose red blood cells carry a fetal form of hemoglobin.
Blood stem cells treated with this gene therapy, transplanted into the mice, engrafted successfully and reduced signs of sickle cell disease.
Other gene therapy trials for sickle cell disease are underway or being developed.
Read more at the Harvard Gazette