Horizon: Trial and ErrorBMJ 2003; 326 doi: https://doi.org/10.1136/bmj.326.7387.506 (Published 01 March 2003) Cite this as: BMJ 2003;326:506
- Alastair Kent, director
- Genetic Interest Group, London
BBC 2, 27 February at 9 pm
The notion of gene therapy—the “overpowering” of a defective gene with a correct copy—is beguilingly simple. For patients with life threatening genetic diseases and for doctors frustrated by their inability to intervene and change the course of the disorder, attacking the underlying biology and dealing with the condition at source is an attractive and potentially effective option. For scientists, the opportunity to work in frontier biology with the possibility of creating platform technologies that may cure disease has inspired some of the brightest and best minds.
But is gene therapy, as this Horizon programme claimed, the “miracle that crashed into tragedy”? The programme told the story of Jesse Gelsinger, a young man with OTC (ornithine transcarbamylase) deficiency. He entered a gene therapy trial in the …