Departmental Papers (HSS)

Document Type

Journal Article

Date of this Version


Publication Source

Perspectives in Biology and Medicine





Start Page


Last Page





In 1989 the gene that causes cystic fibrosis (CF) was identified in a search accompanied by intense anticipation that the gene, once discovered, would lead rapidly to gene therapy. Many hoped that the disease would effectively disappear. Those affected were going to inhale vectors packed with functioning genes, which would go immediately to work in the lungs. It was a bewitching image, repeatedly invoked in both scientific and popular texts. Gene therapy clinical trials were carried out with a range of strategies and occasionally success seemed close, but by 1996 the idea that gene therapy for CF would quickly provide a cure was being abandoned by the communities engaged with treatment and research. While conventional wisdom holds that the death of Jesse Gelsinger in an unrelated gene therapy trial in 1999 produced new skepticism about gene therapy for CF and suggests that CF may provide a particularly compelling case study of a failed genomic technology, perhaps even of a medical "canary." The story of CF might be a find of warning to us that genetic medicine may create as many problems as it solves, and that moving forward constructively with these techniques and practices requires many kinds of right information, not just about biology, but also about values, priorities, market forces, uncertainty, and consumer choice.

Copyright/Permission Statement

Copyright © 2011 The Johns Hopkins University Press. This article first appeared in Perspectives in Biology and Medicne 54:3 (2011), 316-331. Reprinted with permission by Johns Hopkins University Press.



Date Posted: 24 October 2017

This document has been peer reviewed.