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."

Comments

Haemophilia

[rookworm]

the oldest known hereditary bleeding disorder

Aww nuts, I thought it was that other hereditary bleeding disorder.

Re:Haemophilia

[rookworm]

That is merely a symptom, not the disease. (ducks)