Gene therapy rebuilds immunity and its image

Researchers at Great Ormond Street Children's Hospital strengthen the case for gene therapy as a treatment for immunodeficiencies.

Helen Gavaghan(gavers@supanet.com)
Apr 4, 2002

LONDON — There is something about gene therapy that captures the media's and the public imagination, and this week's announcement by Great Ormond Street Children's Hospital in London, that two babies had been treated successfully — not necessarily cured — for a severe, life threatening disease of the immune system was no exception.

Except for similar work undertaken by French researchers in 2000, the public perception of gene therapy has been that the reality did not match the hype and aspiration. In particular, public confidence was shaken in September 1999 when a young man, Jesse Gelsinger, died during a US clinical trial of gene therapy for a liver disease. Though the nature of this trial, being in vivo, was very different from the ex vivo work of the French group and now Great Ormond Street, gene therapy in lay eyes lost some of its shine.

Now, though, the field...

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