Bioethics News

Gene Therapy ‘Cures’ Boy of Blood Disease That Affects Millions

March 1, 2017

(New Scientist) – A TEENAGE boy with an inherited disease that affects millions worldwide seems to have been cured using gene therapy. The treatment appears to have stopped the painful symptoms of sickle cell disease, demonstrating the potential for gene therapy to treat common genetic diseases. The idea of gene therapy – using strands of DNA to compensate for a person’s malfunctioning genes – is almost three decades old. However, the approach has so far mostly been used to treat very rare diseases (see “Long road to success“). In contrast, sickle cell disease affects 100,000 people in the US alone. If the treatment proves successful in larger trials, it could bring gene therapy into widespread use.

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