Gene therapy as an alternative to conventional treatment of haemophilia

A recent experiment showed that a single gene therapy session stimulates the secretion of vital role coagulation factor, offering hope of finding a permanent cure for hemophilia.

Haemophilia is a congenital (inborn), Inherited and characterized by low blood coagulability and therefore, those affected tend to bleed heavily even in a case of minor injuries, in acute cases can occur even spontaneous bleeding.

Currently, there is no cure for this disease, being caused by defects in genes located on chromosome X.

Treatment is usually frequent administration of substances called recombinant factors (VIII or IX, depending on the type of hemophilia, A or B), which compensate coagulation factors that the body can not manufacture itself. Besides involving numerous injections, this treatment is very expensive, and costs could reach hundreds of thousands of dollars per year.

Gene replacement would be the only way to cure hemophilia, and scientists are trying to make it through gene therapy.

Gene therapy involves the use of DNA fragments. These pieces are inserted into the genome of patients with hemophilia by using viruses that play the role of “carriers”. The gene inserted into the viral genome is sent to the liver cell genome and begins to produce the necessary clotting factors.

Researchers at the Institute for the Study of Cancer of the University College London, and the Children’s Hospital St. Jude in Memphis, USA, have addressed this method, using a new virus “carrier”, which aims, specifically, liver cells.

Treatment was tested on six patients suffering from hemophilia. Four of them were able to discontinue therapy with recombinant factors, with no spontaneous bleeding, others two decreased sessions of treatment with recombinant factors, from 2-3 per week to one at 10-14 days.

Two of the patients experienced some degree of deterioration of liver function, which were treated with steroids.

Gene therapy has, according to experts, the potential to cure hemophilia or at least significantly alleviate the symptoms and significantly reduce the suffering of patients as well as costs – often prohibitive – associated with conventional treatment.