News FocusGenetics

The Promise of a Cure: 20 Years and Counting

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Science  19 Jun 2009:
Vol. 324, Issue 5934, pp. 1504-1507
DOI: 10.1126/science.324_1504

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On the 20th anniversary of the identification of the cystic fibrosis gene, as new gene discoveries pile up weekly and hype over the power of genes to transform medicine flows fast, CF offers an object lesson in how difficult it is, and how long it takes, to convert genetic knowledge into treatments. Every CF expert agrees that the gene discovery transformed their understanding of the disease's pathology. But even after so much hard work, not a single therapy based on the CF gene has reached the market. Some promising treatments, especially gene therapy, have proven bitterly disappointing.