Gene Therapy for Hemophilia

Hemophilia is a genetic bleeding disorder where the blood of the patient does not clot during an injury or trauma. This is due to a deficiency or absence of blood clotting protein in the blood. There are twelve clotting factors in the human body. Two blood clotting proteins, a deficiency in either causes hemophilia are factor VIII and factor IX. A deficiency in factor VIII results in hemophilia A while that of factor IX results in Hemophilia B.

Since no permanent cure for hemophilia is available patients have to undergo regular infusions which are painful, expensive and if the infusion is delayed, it can turn out to be extremely damaging in case of a bleed. The therapy alternatives available do provide relief and hemophilia treatments have come a long way over the years but there still is no permanent cure for the condition.

One therapy which could prove to be the magic wand for haemophiliacs is gene therapy hemophilia. Gene therapy involves replacing of the mutated gene with a correct gene to eliminate gene related disorders and treat them at grass root level. Scientists have been working on gene therapy for the past couple of decades and have also achieved success in treating rare genetic disorders

However the first major breakthrough for scientists came last year when they reported successful trials of gene replacement in patients suffering from hemophilia B. This was the first time that a widely occurring genetic disorder had been successfully treated.   Though still in the trial period, gene therapy hemophilia gives hope to all those haemophiliacs who have had traumatic bleeds and subsequent residuals ever since.

Lets us try understanding the process of gene therapy hemophilia in simple terms. It begins with the virus carrying the corrected gene being infused into the body. This virus is called the vector since it is the carrier. It contains corrected gene information. On entering the body this particular virus attacks the liver cells. This is of added importance. Since, clotting factors are synthesized in the liver. Once in the liver, it passes the correct gene information and helps synthesising it.

Amongst the various complications involved in the implementation of this process complication is the body’s immune system. However, the particular virus that was being used currently is completely alien to humans. Our body’s immune system attacks the foreign virus before it can start its corrective action. They hence, have little immunity towards it. By the time the body’s immunity system prepares itself to attack the virus, it already starts its corrective action in the liver.

This therapy can be implemented on one individual only once in his lifetime since, his immune system becomes ready to attack it the second time around. Trials have been carried out for hemophilia B since dealing with factor IX  genes are easier as compared to factor VIII genes.

Gene therapy hemophilia will not only be cost effective for the patients but also give to be a permanent relief once the therapy has been successfully established. Gene therapy hemophilia will give a carefree childhood to the young children suffering from the disease and a normal lifestyle to all those who have grown up with the disease. Now your child would be able to be a part of the soccer team or maybe the school basketball team, with proud bruises to show off as memories. Gene therapy hemophilia makes the haemophiliacs dream of a safer life. Though the therapy has been tried only against hemophilia B, it will soon be implemented in case of patients suffering from hemophilia A too.

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