Gene therapy relieves sickle cell in world first: study
The boy was the first person to be treated, in Paris in October 2014, for sickle cell disease in a clinical trial with gene therapy. The immature cells were treated with a therapeutic gene, carried in a deactivated virus, which recoded their DNA to correct blood cell production. The treated cells were then reinjected into the boy's body. But a team from the AP-HP university hospital group in Paris, the Imagine Institute of Genetic Diseases and gene therapy company bluebird bio said they managed to get a teenager off transfusions. The team collected so-called haematopoietic stem cells, which give rise to red blood cells, from the bone marrow of the youngster, then aged 13.
Gene therapy lets a French teen dodge sickle cell disease
A French teen who was given gene therapy for sickle cell disease more than two years ago now has enough properly working red blood cells to dodge the effects of the disorder, researchers report. "It shows gene therapy is on the right track." Full results have not been reported, but the gene therapy has not taken hold as well in some of them as it did in the French teen. Gene therapy offers hope of a lasting one. About 90,000 people in the U.S., mostly blacks, have sickle cell, the first disease for which a molecular cause was found.collected by :Lucy William