The scientists at Sirius University of Science and Technology (NTU) are determined to develop an innovative gene therapy technology to treat hemophilia B. This groundbreaking research will enable them to design a range of exceptional gene therapy medications to combat various hereditary diseases caused by gene mutations.
Researchers at the Scientific Center for Translational Medicine of the Sirius NTU are developing cutting-edge technology for gene therapy to combat hereditary diseases. They have selected hemophilia B as their model disease. The innovative treatment approaches being pursued are poised to substantially decrease treatment expenses, as well as extend and enhance the quality of life for patients.
It is specified that modern gene therapy drugs are effective, but their cost is extremely high. In addition, it is currently unknown whether the changes made to the genetic code of the patient’s cells will remain for life. If a second injection is necessary, there is a risk of an attack by the body’s immune cells on the drug itself.
Sirius’ scientists intend to solve the problems of previously used approaches to the treatment of hemophilia B, using liposomal nanoparticles instead of viral vectors, where they will place genetic elements, including a DNA editing system based on prime editing and base editing technologies.
“They allow for safe correction of genetic information and provide a long-term therapeutic effect. Such an editing system is capable of making pinpoint, targeted edits of just one nucleotide,” the press service explained.