Genetic engineering for haemophilia A.


At first sight, haemophilia A would appear to be an ideal candidate for treatment by gene therapy. There is a single gene defect; cells in different parts of the body, but especially the liver, produce Factor VIII, and only 5% of normal levels of Factor VIII are necessary to prevent the serious symptoms of bleeding. This review attempts to outline the… (More)


Cite this paper

@article{Gan2006GeneticEF, title={Genetic engineering for haemophilia A.}, author={Shu Uin Gan and Oi Lian Kon and Roy Yorke Calne}, journal={Expert opinion on biological therapy}, year={2006}, volume={6 10}, pages={1023-30} }