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An advance in gene therapy may provide safe treatment to children with a fatal genetic disorder that leads to no functioning immune system.
The majority of children with X-linked severe combined immunodeficiency, or X-SCID, die before the age of one and must live in a "bubble" for protection.
During a clinical trial, nine baby boys were given healthy versions of the faulty gene that codes for the disease.
Eight of the boys were still alive up to 43 months after the treatment.
They have now been able to live a life outside an isolated, sterile room, or a "virtual bubble" of constant medication to try to prevent infections.
The disease affects about one in 250,000 children in the UK.
Scientists in the US, Britain and France led the clinical trial, which built upon an earlier study where a gene was transferred into children with X-SCID.
Safer therapy?It used a virus to the transfer the gene, but it also activated other genes that led to leukaemia in five out of the 20 patients.
Scientists changed the virus so...