Gene therapies based on in vivo ribonucleic acid transfer
Medical Review (Med. pregled), 2025, 61(3), 60-68.
T. Vekov1, L. Dimova1, N. Chilingirova1, V. Belcheva2
1 Heart and Brain Hospital – Pleven
2 Trakia University – Stara Zagora
Abstract. RNA therapies include a wide range of strategies and mechanisms of action, because there are different types of RNA that have diverse functions in the cell – mRNA vaccines and immunotherapies, gene therapies based on miRNA, gene silencing by siRNA, therapies for monogenic diseases by asRNA, RNA aptamers for gene therapies. As of 2024, 20 drug therapies approved for clinical use based on in vivo ribonucleic acid transfer are available. The aim of the study is to analyze genetic engineering technologies for RNA modification in order to better understand the strategy for developing and applying therapies based on in vivo RNA transfer. Documentary methods were used to analyze published scientific data from preclini¬cal and clinical trials of gene therapies based on RNA transfer. The mechanism of action and strategies for modifying RNA, as well as their clinical application in modern therapies for the treatment of various diseases were investigated.
Key words: gene therapies, RNA, in vivo transfer, mechanisms of action
Address for correspondence: Prof. Toni Vekov, PhD, DSc, e-mail: