But when the 18-year-old Jesse Gelsinger died during a clinical trial for a revolutionary new gene therapy technique in 1999, that bubble of expectation burst. The mantle of medical hyperbole – and the funding – moved on to other, as yet untarnished advances, including stem cells and the promise of personalised medicine.

Although investment in gene therapy withered, away from the limelight research has continued and in the intervening years a number of important breakthroughs and treatments have been developed. The latest of these, described in a paper published in the journal Nature, may go some way towards reinstating gene therapy in medical science’s premier league.

Scientists at the Wellcome Trust’s Sanger Institute and the University of Cambridge have succeeded in correcting the genetic mutation responsible for Antitrypsin deficiency – a debilitating condition that causes cirrhotic liver disease and lung emphysema in some 30,000 patients in the UK alone.
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Bringing gene therapy in from the cold – BBC News