The Promise of a Cure: 20 Years and Counting


June 22, 2009

Science Magazine

Science magazine covers what has happened at the bench and in the clinic in the 20 years since the discovery of the gene for cystic fibrosis. 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, no therapies based on the CF gene have reached the market. Although gene-therapy approaches haven't paid off, prospects have improved for those with CF: Their median life expectancy has stretched almost 10 more years and now exceeds 37. James Wilson, MD, PhD, professor of Pathology and Laboratory Medicine, comments on the attitude among gene therapists in the fall of 1990, a year after the CF gene discovery: "Expectations for the success of gene therapy for CF were as high as I've ever seen for any disease, under any circumstances, in the 20 years I've been involved in this." Read More (full article access may be restricted)