This study focuses on gene therapy as a biotechnological and medical approach for modifying the genome of human somatic cells. The most widely used method for gene delivery is recombinant adeno-associated viral vectors (rAAV). The study aims to achieve high-copy integration of the gene of interest (GOI) in a stable cell line. The Sleeping Beauty transposon system and lentiviral vectors (rLV) were tested. The results demonstrated that cell pools obtained via rLV exhibited low rAAV titers, presumably due to the structure of the rAAV transfer cassette. The Sleeping Beauty system was found to be the most efficient, leading to two monoclonal cell lines capable of stable rAAV production over 30 passages. Further optimization through additional rounds of GOI integration is being considered.

Григорьев Я.А. (науч. рук. Перепелкина М.П., Радько С.В.) Integration of transgenes for the establishment of an aav-producing cell line // Сборник тезисов докладов конгресса молодых ученых. Электронное издание. – СПб: Университет ИТМО, [2025]. URL: https://kmu.itmo.ru/digests/article/15152