Haemophilia is a group of hereditary genetic disorders that impair the body's ability to control blood clotting or coagulation, which is used to stop bleeding when a blood vessel is broken. Treatment to this disorder is usual costly, thus research on a new protein treatment is needed to solve this problem.
A research team at the Children's Hospital in Philadephia, USA found that a custom built variety of coagulation factor Xa (FXa) was effective in controlling and clotting bleeding in haemophilliac mice. It appeared to do so more safely, and with greater long term prospects.
Traditionally, haemophilliacs require fairly regular infusions of clotting proteins in order to avoid uncontrolled bleeding.
Often, the body perceives these proteins as foreign organisms that need to be eliminated, which creates an immune response that more or less makes protein treatment useless.
At this point, the only other option for sufferers is spend a small fortune on drug treatments instead, and even this isn't guaranteed to work.
The thrust of this new treatment, according to the study team, is to take the naturally occurring Fxa and reshape the actual protein into a customised shape, which not only means that it stays in the system for long and requires a lesser quantity of protein, but it also means doctors can much more easily control the way the protein interacts with other physiological systems, making it more predictable and safe to use that the unpredictable natural FXa.
"Our designed variant alters the shape of FXa to make it safer and efficacious compared to the [naturally occurring] wild-type factor, but much longer-lasting in blood circulation," said the head researcher.
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