Named after scientists from the First Moscow State Medical University. Sechenov The Russian Ministry of Health is developing a gene therapy drug based on viral vectors for the treatment of Usher syndrome. socialbites.ca was told about this at Sechenov University.
Usher syndrome is a genetic disease characterized by progressive hearing and vision loss. First, the person’s vision weakens in low light, they lose the ability to navigate at dusk, and then peripheral vision is lost. The disease continues to progress and gradually leads to complete blindness. There is no medicine yet to stop this process, neither in Russia nor in the world.
The development of retinal degeneration in Usher syndrome is associated with the mutation of a specific gene. The developed drug stops the development of the disease by delivering a copy of the gene disrupted in Usher syndrome to the retina cells, thanks to viral vectors.
“Our laboratory develops and tests gene therapy drugs based on viral vectors. As viral vectors, we use adeno-associated viruses that are safe for humans. They do not multiply in the body and do not cause disease, they only transfer genetic material into cells,” the head of the laboratory, Alexander Malogolovkin, told socialbites.ca. They are a transmitting system.
Researchers have already developed the technology to produce viral vectors, purify and concentrate them, and have also created a unique genetic structure and “packaged” it into viral vectors. The development was tested on cell culture of the retinal pigment epithelium under laboratory conditions. The result showed the possibility and functionality of the delivered copy of the gene to be delivered into cells. The next phase is testing on laboratory animals, which scientists plan to start in early 2024.
“Our gene therapy drug will not restore vision to patients who have already lost their vision. Our aim is to develop a drug that will slow the progression of blindness in Usher syndrome. We also hope that such a treatment could completely stop vision loss in people who do not yet show symptoms of this genetic disease, because what happens to such a treatment in the future The earlier it is started, the better the chances of seeing it,” Malogolovkin emphasized.
Previously in Russia was created “Artificial muscles” powered by alternating current.
Source: Gazeta
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