People with Haemophilia A have a dangerous bleeding disease. People get it when their bodies don’t have enough of a protein called Factor VIII. This protein helps blood clot. This can cause bleeding out of the blue, which makes life very hard for those who are impacted. The number of people with this problem in India is about 136,000, which is the second highest number in the world.
Breakthrough in Gene Therapy
Indian experts have made a big progress in the fight against haemophilia A. Using lentiviral vectors, scientists at the Centre for Stem Cell Research (CSCR) in Vellore have made a new gene treatment. This therapy was tried on people for the first time in India. The study included five individuals ages 22 to 41 years. The findings were very positive. After the treatment, none of the five people who took part in it bled again during the next year. They also made Factor VIII for a long time without having regular infusions. This is a big improvement over current treatments, which can be expensive and tough for patients, especially children.
Long-Term Results and Hope for the Future
The subjects were followed for six months after getting the gene therapy. The researchers found a strong link between the amounts of Factor VIII in the blood and the number of gene copies introduced into their cells. This shows that the medicine is not only successful but also safe for long-term use. This new gene treatment gives hope for many people suffering from severe haemophilia A. It could change their lives by lowering the need for regular treatments and improving their quality of life. The success of this study is a major achievement for Indian science and medicine, paving the way for better medicines for haemophilia and probably other genetic diseases in the future.
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