Scientists at the Laboratory of Molecular Virology at Sechenov First Moscow State Medical University have created a new drug for the treatment of Usher syndrome. This is a hereditary disease that leads to blindness.
Doctors conducted tests on transgenic mice. It turned out that with the use of the drug, blindness in rodents develops more slowly.
The drug is based on viral vectors that deliver copies of the gene into retinal cells. Scientists have confirmed the effectiveness of the technology on a cell culture.
It is worth mentioning that Usher syndrome type II and Stargardt disease are orphan diseases. They are rare but seriously affect the quality of life and can lead to disability in working age.
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