Scientists
have used gene therapy to relieve the symptoms of a teenager suffering from
sickle cell disease (SCD) in a world-first breakthrough, it has been reported.
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 worldwide -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 per son to be treated, in Paris in October 2014, for SCD in a
clinical trial with gene therapy. The team collected socalled haematopoietic
stem cells, which give rise to red blood cells, from the bone marrow of the
youngster, then aged 13. 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.
Source: TIMES OF INDIA-7th March,2017
