What next for human gene therapy?
By Eric T. Juengst,
British Medical Journal
| 06. 28. 2003
The high hope of genetic medicine for 30 years has been to develop a way of using recombinant DNA techniques to treat patients through the genes involved in their diseases. As Richard Roblin, scientific director of the Council on Bioethics of the President of the United States, put it in 1979: "There is something aesthetically compelling about cutting to the heart of the problem, by treating the disease at the molecular level, where it originates."1 Since 1990, this vision has generated a modest industry of bench research and animal studies, culminating in almost 1000 clinical trials in humans around the world, for a wide variety of diseases.2 In the past few years, however, the field has learned that in genetic medicine, as in war, the "surgical strike" is rarely as clean and effective as theory implies it should be.
After almost a decade without much clinical success,3 the field has experienced in quick succession its first iatrogenic death,4 its first apparent "cures,"5 and then among those cured patients the first instances of serious downstream disease traceable to the main theoretical...
Related Articles
By Mustapha Bature Sallama, Modern Ghana | 06.11.2026
In much of West Africa, a woman who cannot bear children does not merely face a medical condition. She faces a verdict. Her marriage may unravel. Her community may turn cold. Her identity, in a social order that ties womanhood...
By Emily Baumgaertner Nunn, The New York Times | 06.11.2026
When scientists at Columbia University announced they had used a newer technology to precisely edit the genes of human embryos last week, they set the academic community ablaze with debate. Is this good news or bad? How fast will...
By Gina Kolata, The New York Times | 06.11.2026
On my second visit with Nancy Wexler at her Manhattan apartment, she had a gift for me. It was a copy of her newly published memoir, “My Life, My Science: Pursuing a Cure for Huntington’s Disease.”
It had been signed...
By Robert P. Baird, The Guardian | 06.30.2026
In 2017, a 33-year-old political philosopher named Iason Gabriel was told by a friend that he ought to apply for a job at DeepMind, the London-based subsidiary of Google where much of its AI research was concentrated. The suggestion was not...
