Gene therapy: Ingenious

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UK Only Article:  standard article




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The worldwide wobble




Fly Title: 


Gene therapy





Location: 


New York




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20140208_STD001_0.jpg








IT SOUNDS like science fiction, and for years it seemed as though it was just that: fiction. But the idea of gene therapy—introducing copies of healthy genes into people who lack them, to treat disease—is at last looking as if it may become science fact.
The field got off to a bad start, with the widely reported death of an American liver patient in 1999. In 2003 some French children who were being treated with it for an immune-system problem called SCID developed leukaemia. Since then, though, things have improved. Indeed one procedure, for lipoprotein lipase deficiency (which causes high levels of blood fats, with all the problems those can bring), has been approved, in Europe, for clinical use.

The most recent success, announced last month in the Lancet, was of an experimental treatment for choroideremia, a type of blindness. This is ...