The Sirius University of Science and Technology will develop a gene therapy technology for hemophilia B. This was reported by the university's press service.
The development will form the basis for creating new drugs for the therapy of hereditary diseases.
Scientists have chosen hemophilia B as a model disease. New treatment strategies will significantly reduce the cost of treatment and prolong and improve the quality of life for patients.
Scientists plan to change the approach to treating hemophilia B. Instead of viral vectors, it was decided to use liposomal nanoparticles. They will contain genetic elements and a DNA editing system based on prime editing and base editing technologies.
They allow for the safe correction of genetic information and ensure a long-term therapeutic effect. Such an editing system is capable of making pinpoint, targeted edits of only one nucleotide in size.
It is worth noting that specialists from the Sirius University previously developed a new method for diagnosing age-related and oncological diseases by measuring vitamin B12 in human cells.
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