Led by Amit Nathwani at University College London, scientists introduced the working gene for FIX to patients by injecting them with cold viruses that were engineered to carry the gene in, infect cells, and start producing the needed clotting protein. This is the standard technique used in gene therapy, but often it fails because the immune system kills off the cold viruses before they can do their work.
Nathwani's team was able to figure out a more efficient way to install the new genes. At the start of the study, all of the participants had less than 1% of normal levels of FIX in their blood. By the end of the study, which followed patients for as long as six to 16 months, most had FIX levels that were 3% to 11% of normal values. Two patients each received low, intermediate or high doses of the gene therapy in an IV-like infusion in the arm.
New England Journal of Medicine - Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B
Haemophilia B (factor IX deficiency) occurs in around 1 in about 20,000–34,000 male births.
Haemophilia is rare, with only about 1 instance in every 10,000 births (or 1 in 5,000 male births) for haemophilia A and 1 in 50,000 births for haemophilia B. About 18,000 people in the United States have haemophilia. Each year in the US, about 400 babies are born with the disorder. Haemophilia usually occurs in males and less often in females. It is estimated that about 2500 Canadians have haemophilia A, and about 500 Canadians have haemophilia B
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