Gene Therapy Corrects Hemophilia in Mice and Dogs

FiReaNGeL writes "Researchers successfully corrected hemophilia, the oldest known hereditary bleeding disorder, in dogs and mice. They introduced the gene coding for clotting factor VIII, which is defective in type A hemophilia, in the liver of newborn animals. All animals achieved expression of the clotting factor, averaging 139 and 115 percent of normal factor VIII activity and are stable after more than a year. The treatment targeted newborns exclusively for two reasons; their liver is still developing and their immune system is immature, facilitating the gene transfer. Researchers plan to perform tests in higher primates in the next few years. A summary of the research and the reference to the original paper are available."

Re:Speaking as a hemophiliac (type B)...

[Xenkar]

Actually the Benefix Recombinant Factor products are made from genetically altered hamsters. The process of harvesting the factor kills the rodents so my existence is the bane of any animal rights advocates.

This also means that I won't get HIV or any other nasty blood disease. Most hemophiliacs don't feel safe using donor blood due to the HIV outbreaks of the Early 80's. About 44% of hemophiliacs at the time were infected.

The blood supply is supposedly perfectly safe now, but I wouldn't touch that stuff even if it'd save my life.

My uneasiness comes from the thought that someone could get infected with HIV and go donate blood before any detectable amount of HIV anti-bodies form in the blood. They actually test for the presence of the anti-bodies instead of the actual virus.

As a Mild Haemophiliac

[khanyisa]

Spelling it nicely in British English :-) Not all Haemophilia is as severe as mentioned in the article, I have 14% of the normal clotting factor and haven't had a problem for years... of course a head injury etc is still a major risk. But this is really nice research to see