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marilynaliza | healthiswealth

Gene therapy of hemophilia

Jun 16th 2014 at 10:28 PM

Hemophilia B is a genetically determined disease in which the production of so-called impaired. Factor IX which is an essential element of proper blood clotting. To date, the treatment of hemophilia B consisted primarily of factor IX administration by injection,

sometimes several times a week. Now, thanks to the collaboration of researchers from University College London and St

. Jude Children's Research Hospital, appears in the hope of creating a method of treatment based on gene therapy , giving long-lasting effects.

The researchers used in their work virus particle that infects humans it is true, but not giving any symptoms. Modified virus that transferred genetic material factor IX into the liver of patients with hemophilia B, and started its production

The first tests took part in 6 patients, of whom two received the low dose of virus, two middle and two high dose.

Typically, patients with hemophilia activity of factorIX is less than 1% of the activity in healthy people. Meanwhile, the use of gene therapy in patients participating in the experiment the factor IX activity was observed from 2 to 12%, and this level was maintained for up to several months.

"I did not need their current treatment, both in prophylaxis and in case of injuries. Previously, I had to do injections at home three times a week.

"- Says one of the patients -" I play soccer, run, take part in triathlon and before I had to poke before and sometimes after that. The fact that I do not have it anymore is wonderful. "

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