Gene therapy is a treatment method where necessary DNA is delivered to cells to help the body fight disease. Special safe "courier" viruses are used for this: they do not cause illness but transport therapeutic material directly into the cell. Researchers at the Scientific Center for Translational Medicine of NTU "Sirius" have found a way to make such a virus almost twice as effective.

The development is based on changing the assembly method of the AAV9 virus. Typically, its shell consists of three types of proteins: VP1 and VP2 help the virus penetrate the cell and deliver material to the nucleus, while VP3 forms a protective shell. The problem is that with conventional production, there are fewer VP1 and VP2 proteins inside the virus than needed for maximum efficiency.

Scientists at the Scientific Center for Translational Medicine of NTU "Sirius" found a way to overcome this limitation. They added an additional, fourth, genetic element to the virus growth mixture. As a result, there were more VP1 and VP2 proteins inside the shell, and the "courier" virus began to work significantly more efficiently. Laboratory tests on human cell cultures showed that the new viral particles penetrated cells 1.9–2.1 times better than conventional ones, while the shape and size of the viruses remained unchanged.

According to the first author of the study, Maxim Efremov, the new technology does not require expensive equipment or a complete restructuring of production. To enhance the work of the "courier" virus, it is enough to change the ratio of components in the mixture for its cultivation. This will make the method easier to implement in pharmaceutical production.

Currently, more than a hundred AAV-based drugs are undergoing clinical trials worldwide, and seven have already been approved by regulators. In the future, scientists plan to scale the technology and test it on models of retinal and nervous system diseases.