Scientists 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. SCD is an inherited disease caused by a gene mutation that results in red blood cells losing their usual donut-like appearance and taking on a sickle or crescent moon shape.
Sufferers - around five million world-wide - often have anaemia and get tired easily, run a higher risk of infections and stroke, and experience bouts of severe body pain.
Many need chronic blood transfusions. 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 boy was the first person to be treated, in Paris in October 2014, for sickle cell disease in a clinical trial with gene therapy. Others have been tested since, but no official results published. 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.
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