Hemaphilia is a serious blood related disease. Actually, in this disease, if a person starts bleeding then it does not stop. If a person gets this disease, his blood does not clot quickly. Hemophilia B is caused by mutations in the gene for coagulation factor IX. It is a protein that helps in blood clotting. People with missing or low levels of factor IX bleed longer than healthy people.
Whereas people with mild forms of the disorder experience excessive bleeding only after an injury. About 40-50% of patients have a more severe form that causes spontaneous internal bleeding. Which can cause serious joint damage and even death. The primary treatment for people with severe hemophilia B is injections of factor IX every 3 to 4 days for the rest of their lives. Which is often a very long and expensive treatment.
Treatment of hemophilia through gene therapy
Researchers from St. Jude Children’s Research Hospital, University College London and the Royal Free Hospital have pointed towards gene therapy for the treatment of this disease. This approach takes advantage of viruses that infect humans but do not cause disease, called vectors. When the human factor IX gene is inserted into these vectors. So viruses deliver genes to the cells they infect. The cells then make functional proteins. Previous studies had some success using such a vector to target liver cells that produce human factor IX. However, achieving stable long-term expression of factor IX was a challenge. Because it complicated liver toxicity.
According to a new research, human factor IX gene was inserted into an improved vector recently developed for gene therapy. Which is called adeno-associated virus serotype 8 (AAV8). Ten men aged 22 to 64 years participated in the study. Two were given low doses of the vector through a peripheral vein. 2 were given medium dose and 6 were given high dose. The study was supported in part by the NIH Heart, Lung, and Blood Institute (NHLBI). The results were published in the New England Journal of Medicine on November 20, 2014.
Within 4 months of receiving the modified gene therapy, the levels of factor IX activity in patients’ blood increased from less than 1% of normal to between 1% and 6% of normal. People who were given higher doses of vector. They produced higher levels of clotting proteins. The higher dose resulted in a substantial reduction in the incidence of bleeding as well as less need for treatment with factor IX.
These improvements lasted for the entire observation period. Which was up to 4 years for some participants. Side effects were mild. The most common adverse effect was seen in 4 out of 6 men who received the higher dose of the virus. He had increased levels of liver enzymes. Which is a sign of liver inflammation which can be easily treated.
What is hemophilia?
Hemophilia is a blood disorder. There is no blood clot in it. There is a danger that once there is an injury or cut, bleeding will start and will not stop. If a person has this disease, it means that his body does not have the necessary protein which forms blood clot. Blood clotting proteins which combine with platelets and form blood clots are not present. Due to which blood starts coming out without stopping.
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The most surprising thing is that India is at second place in terms of the number of hemophilia patients. There are about 1.3 lakh patients in India.
Which people have this problem?
For your information, let us tell you that this problem is more common in boys than girls. Boys have one X chromosome. If bad chromosomes are passed on to the child from the mother, then this disease starts developing in the child. Girls have 2
Disclaimer: Some information given in the news is based on media reports. Before implementing any suggestion, you must consult the concerned expert.
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