Hemaphilia is a serious blood-related disease. In fact, in this disease, if a person starts bleeding, it does not stop. If a person gets this disease, their blood does not clot quickly. Hemophilia B is caused by mutations in the clotting factor IX gene. It is a protein that helps blood clot. People with low or absent factor IX levels bleed longer than healthy people.
While people with mild forms of the disease experience excessive bleeding only after an injury. Around 40 to 50% of patients have a more severe form causing spontaneous internal bleeding. This can cause serious joint damage or even death. The main treatment for people with severe hemophilia B is factor IX injections every 3 to 4 days for the rest of their life. Which is often a very long and expensive treatment.
Treatment of hemophilia with gene therapy
Researchers from St. Jude Children’s Research Hospital, University College London and the Royal Free Hospital have indicated 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. Viruses therefore transmit genes to the cells they infect. The cells then produce functional proteins. Previous studies have had some success using such a vector to target liver cells that produce human factor IX. However, achieving long-term stable expression of factor IX was challenging. Because it complicated liver toxicity.
According to new research, the human factor IX gene has been inserted into an improved vector recently developed for gene therapy. This is called adeno-associated virus serotype 8 (AAV8). Ten men aged 22 to 64 participated in the study. Two of them received low doses of the vector through a peripheral vein. 2 received a medium dose and 6 a high dose. The study was funded 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 the modified gene therapy, levels of factor IX activity in patients’ blood increased from less than 1% of normal to between 1% and 6% of normal. People who received 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 a reduced need for factor IX treatment.
These improvements lasted throughout the observation period. Which could go up to 4 years for some participants. Side effects were mild. The most common side effect was seen in 4 out of 6 men who received the highest dose of the virus. He had increased liver enzyme levels. This is a sign of liver inflammation that can be easily treated.
What is hemophilia?
Hemophilia is a blood disease. There’s no blood clot in it. There is a risk that once there is a wound or cut, bleeding will start and not stop. If a person suffers from this condition, it means that their body does not have the proteins necessary for the formation of the blood clot. Blood clotting proteins that combine with platelets to form blood clots are not present. This is why the blood starts flowing without stopping.
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The most surprising thing is that India ranks second in terms of the number of hemophilia patients. There are around 1.3 lakh patients in India.
Which people have this problem?
For information, let’s say that this problem is more common in boys than in girls. Boys have one X chromosome. If bad chromosomes are passed to the child from the mother, this disease begins to develop in the child. Girls have 2 X chromosomes, in such a situation they are less likely to get this disease.
Disclaimer: Some information given in news is based on media reports. Before implementing any suggestion, you should consult the relevant expert.
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