Gene therapy shows promise for haemophiliaBMJ 2001; 322 doi: https://doi.org/10.1136/bmj.322.7300.1442/a (Published 16 June 2001) Cite this as: BMJ 2001;322:1442
- Scott Gottlieb
- New York
A new type of gene therapy boosted levels of factor VIII clotting factor in a small number of people with haemophilia, allowing two severely ill patients to go for 10 months without spontaneous bleeding, a new study says.
These results are far from the proof needed to bring the treatment to market. In particular it is not clear if the effect lasts long …