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A pioneering gene therapy for sickle cell disease is working well so far for a 13-year-old French boy with the hereditary blood disorder, researchers said on Saturday, in a boost for the technology to fix faulty genes.
He is the first patient with severe sickle cell disease (SCD) to be treated with Bluebird Bio's LentiGlobin BB305 product, which the U.S. biotech company believes could cure the disorder.
SCD is caused by a mutated gene, resulting in abnormal red blood cell function. Patients suffer anemia, painful obstruction of blood vessels and, in some cases, early death.
Bluebird's gene therapy treats the condition by extracting blood stem cells and then adding a working version of the malfunctioning gene.
Results presented at the European Hematology Association meeting in Vienna showed the French patient has not needed a life-sustaining blood transfusion for more than three months and his body was producing 45 percent so-called anti-sickling hemoglobin at six months.For more click here
source: Medical Daily