Teenager's sickle cell reversed with world-first therapy according to : BBC

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.




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 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. 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.

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.



collected by :Lucy William

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