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Five children with a genetic disease that wipes out their immune system have successfully been treated with gene therapy

Editorial: "Gene therapy needs a hero to live up to the hype"

MOST parents dream of a 5-week-old baby who sleeps through the night, but Aga Warnell knew something was wrong. Her baby, Nina, just wasn't hungry in the way her other daughters had been.

Within weeks, Nina became very ill, says her father, Graeme. She was admitted to hospital with a rotavirus infection. Then she picked up pneumonia.

It turned out Nina had a condition called severe combined immunodeficiency (SCID). She had been born without an immune system due to a genetic defect. It is also known as "bubble boy" disease, since people affected have to live in a sterile environment. "The doctors said 'you need to prepare yourself for the fact that Nina probably isn't going to survive'," says Graeme.

A year-and-a-half later, Nina is a happy little girl with a functioning immune system. She has gene therapy and its latest improvements to thank for it.

SCID was the first condition to be treated with gene therapy more than 20 years ago. A virus was used to replace a faulty gene with a healthy one. But in subsequent trials, four young patients were diagnosed with leukaemia two years after receiving a similar treatment. An 18-year-old also died following a reaction to a virus used in gene therapy for a liver condition. It was the start of a rocky road (see "Trials and tribulations of gene therapy").

Gene therapy has come a long way since, and Nina's case, along with others, mark a turning point: researchers seem to have found a safer way of manipulating our genes.

Preliminary results for the first two children to receive the improved SCID gene therapy 18 months ago were presented at the European Society of Gene and Cell Therapy conference in Madrid, Spain, last week. The children's immune systems have continued to improve since receiving the treatment, says Bobby Gaspar of Great Ormond Street Hospital in London, who led the trial.

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'Bubble kid' success puts gene therapy back on track

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