Scientists have slowed the development of hereditary blindness in lab mice

The Laboratory of Molecular Virology at the I.M. Sechenov First Moscow State Medical University is developing an innovative gene therapy drug for the treatment of Usher syndrome, an inherited disease of the retina in which a person gradually loses his or her vision. The researchers tested the drug on laboratory transgenic mice and obtained the first encouraging results.

"Together with our colleagues from the RetinaFoundation for research and treatment of retinal diseases, the interregional public organization "To See" and ophthalmogenetic doctors, we have identified two hereditary retinal diseases for our further research. These are Usher syndrome type 2 and Stargardt's disease. Both diseases are categorized as orphan diseases. However, these pathologies lead to disability of patients, and at working age", - said the head of the laboratory Alexander Malogolovkin.

The researchers worked out the technology of viral vectors production, their purification and concentration, as well as created a unique genetic construct and "packed" it into viral vectors. Earlier the development was tested in laboratory conditions on cell culture of human retinal pigment epithelium. The result confirmed the ability of viral vectors to deliver copies of the gene into cells. And now another important stage of the research has been completed - the new technology of genetic material delivery to retinal cells was tested on laboratory transgenic animals.

The experiment showed that in mice, to which the gene therapy drug was delivered, it was possible to slow down the development of degenerative changes in the retina in Usher syndrome.

Read more in an exclusive Izvestia article:

Vector of change: scientists have made a breakthrough in the treatment of hereditary blindness