as declared in 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.
as mentioned in Researchers are reporting early success using gene therapy to treat, or even potentially cure, sickle cell anemia.
Medical experts stressed, however, that much more research lies ahead before gene therapy can become an option for sickle cell anemia.
Williams and his colleagues are studying a different approach to sickle cell gene therapy.
It’s a high-tech treatment, and many sickle cell patients are low-income and far from a major medical center, he said.
That’s a big change from his situation before the gene therapy, according to his doctors at Necker Children’s Hospital in Paris.
as mentioned in
Gene therapy offers new hope for healing sickle cell disease
Gene therapy offers new hope for healing sickle cell diseaseIn a world first, this blood disease which afflicts babies has been successfully treated by gene therapy.
For the first time ever, a patient suffering from sickle cell disease has been successfully treated by means of gene therapy.
In a world first, a patient with sickle cell disease has been successfully treated by gene therapy in the Necker Hospital in Paris.
“And gene therapy can then offer a solution.”In October 2014, Professor Cavazzana’s team applied the treatment to a 13-year-old adolescent suffering from severe sickle cell disease.
“However, there is every indication that his condition will remain stable.”Sickle cell disease is particularly frequent among people from the Antilles, Africa, and the Mediterranean.
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