Scientists: gene therapy successfully cured the «illness of the Tsarevich Alexei»

© Fotolia / psdesign1Модель DNA moleculesScientists: gene therapy successfully cured the «illness of the Tsarevich Alexei»© Fotolia / psdesign1

Genetics from Britain, announced the successful completion of the first phase of clinical trials of gene therapy that redeems man from the classic form of hemophilia, affecting crown Prince Alexei and many other descendants of the British Queen Victoria, said in an article published in the journal NEJM.

«The results of the experiments were so stunning that they exceeded our wildest expectations. When we started the experiments, we thought that even a 5% improvement will be a great success, and therefore full restoration of the blood clotting factor and a sharp decrease in force and frequency of bleeding pleasantly surprised us. It radically transforms the lives of people suffering from this disease,» said John Pasi (Pasi John) from Queen Mary University in London (UK).

Hemophilia is one of the most well-known hereditary diseases, which affects only men, while women can only be carriers of the disease gene. In the relatively recent past it was considered a «disease of the noble people», since she suffered the descendants of many of the monarchs and to know, for example, the descendants of the British Queen Victoria Tsarevich Alexei, son of Nicholas II.

The most common type of hemophilia — hemophilia a, is associated with a mutation in which the body does not produce protein F8, coagulation factor VIII, causing the blood can not clot the appearance of cuts and bruises. With the advent of stem cell and gene therapy, scientists have a real opportunity to create a vaccine from hemophilia, or at least to suppress the unpleasant consequences of developing the disease.

The first version of such a gene therapy came about four years ago, and then scientists have successfully tested it on dogs, primates and other laboratory animals. Clinical trials, which involved nine volunteers with severe forms of hemophilia started in five cities of Britain in September 2015.

In these experiments, as noted biologist, was attended by nine young people, repeatedly experiencing heavy bleeding and other problems associated with the lack of «normal» blood coagulation factor in their body. They agreed that the scientists will put into their body is a special virus that will stick in their DNA «normal» version of the F8 gene. About half of the volunteers received a fairly small and safe doses of the virus, and the other is a very large amount.

According to Pasi, the experiment ended a very successful way – 85% of the volunteers got rid of all symptoms of hemophilia few weeks after infection, and all the participants of the experiments has significantly improved their health. As the researchers note, more than half of their wards completely abandoned injection F8, and their condition remained stable over the past year.

«Gene therapy has completely changed my life, now I have hope. Now I can play with my kids in football, with them climbing trees and doing all those things that attract students and Teens. I used to be scared of the idea, if I can walk when I’m 40. At 23, I couldn’t run 100 meters to catch the bus. I am now 29 and I walk at least two miles during everyday walks,» says Jake Omer (Omer Jake), one of the participants in the experiments.

Source