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Gene therapy lets a French teen dodge sickle cell disease

as mentioned 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.

 

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As informed in 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.
Great hope has been raised concerning the treatment of a blood disease affecting some 250,000 babies at birth each year around the world.
Two years after undergoing treatment, the patient is in complete remission.
This breakthrough, which was announced on Thursday in an article in the New England Journal of Medicine, was achieved by a team from the Necker Hospital in Paris and the Imagine Institute (1).

Gene therapy relieves sickle cell in world first: study

as informed in By AFPMore by this AuthorScientists have used gene therapy to relieve the symptoms of a teenager suffering from sickle cell disease (SCD) in a world-first breakthrough, they reported on Thursday.
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 team collected so-called haematopoietic stem cells, which give rise to red blood cells, from the bone marrow of the youngster, then aged 13.
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.

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