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Doctors reverse teen’s sickle cell disease with innovative gene therapy

according to Sickle cell disease is a severe hereditary form of anemia, which causes patients to develop abnormal hemoglobin in red blood cells.
The procedure, which was performed at Necker Children’s Hospital in Paris, may offer hope to millions of patients who suffer from sickle cell disease, BBC News reported.
In a world first, doctors at Necker Children’s Hospital removed his bone marrow and genetically altered it using a virus to compensate for the defect in his DNA responsible for sickle cell disease, BBC News reported.
A French teen who underwent a first-of-its-kind procedure 15 months ago to change his DNA shows no signs of the sickle cell disease he had been suffering from.
Before undergoing the procedure, treatment for the unidentified teen included traveling to the hospital each month for a blood transfusion to dilute the defective blood, BBC News reported.

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as informed in Sickle cell disease causes normally round red blood cells, which carry oxygen around the body, to become shaped like a sickle.
But the study does show the potential power of gene therapy to transform the lives of people with sickle cell.
Image copyright SPL Image caption Healthy red blood cells are round, but the genetic defect makes them sickle shapedA French teenager’s sickle cell disease has been reversed using a pioneering treatment to change his DNA.
Image copyright Science Photo LibraryWhat is sickle cell disease?
However, the expensive procedure can only be carried out in cutting-edge hospitals and laboratories, while most sickle cell patients are in Africa.

as informed in

One boy’s cure raises hopes and questions about gene therapy for sickle cell disease

“It’s a bit of a megaphone, allowing us to say that gene therapy might lead to a dramatic outcome.”
Aclosely watched study using gene therapy to treat sickle cell disease cured one patient, a boy in France, researchers reported on Wednesday, a glimmer of hope for a long-neglected disease but one that comes with several caveats.
The new paper, in the New England Journal of Medicine, provides a “deeper view” of the patient’s disease and recovery, said Bluebird CEO Nick Leschly.
Results from the clinical trial, which is sponsored by Cambridge, Mass.-based Bluebird Bio, have been dribbling out at scientific meetings and in company announcements since soon after the boy received the gene therapy, in October 2014, when he was 13.

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