Somatic gene therapyBMJ 1996; 312 doi: https://doi.org/10.1136/bmj.312.7027.323 (Published 10 February 1996) Cite this as: BMJ 1996;312:323
- Julia Dorin
- Scientist MRC Human Genetics Unit, Western General Hospital, Edinburgh EH4 2XU
Optimism tempered by reality
Treating diseases by putting recombinant genes into somatic cells has been heralded as a brave new way forward for molecular medicine. The appeal of this strategy for inherited gene disorders is its simplistic rationale—that introducing a normal copy of the defective gene into mutant cells will ameliorate the disease phenotype. This means in theory that correcting the fundamental abnormality will treat both immediate and later symptoms even when the underlying biology of the disease may not be completely understood. It is thus understandable that gene therapy has been regarded with huge optimism. However, the absence of sensational cures now puts such optimism in question. Has there been any real progress towards clinical application, and is it realistic to expect single gene disorders such as cystic fibrosis, familial hypercholesterolaemia, and adenosine deaminase deficiency to be cured?
Diseases that offer the best prospects for gene therapy are those caused by single recessive gene defects. Of these, perhaps the highest priority for research should go to those that are fatal and in which the gene responsible is cloned, the target organ is relatively accessible, current treatments are inadequate, and there is some evidence from laboratory and animal studies that the suggested approach will be safe and effective. Scientific kudos and venture capital are certainly driving the move into clinical trials using first generation vectors and protocols; and the first …