Shtykalova Sofia, Deviatkin Dmitriy, Freund Svetlana, Egorova Anna, Kiselev Anton
Department of Genomic Medicine, D.O. Ott Research Institute of Obstetrics, Gynecology and Reproductology, Mendeleevskaya Line 3, 199034 Saint-Petersburg, Russia.
Faculty of Biology, Saint-Petersburg State University, Universitetskaya Embankment 7-9, 199034 Saint-Petersburg, Russia.
Life (Basel). 2023 Mar 29;13(4):903. doi: 10.3390/life13040903.
Over the past decades, non-viral DNA and RNA delivery systems have been intensively studied as an alternative to viral vectors. Despite the most significant advantage over viruses, such as the lack of immunogenicity and cytotoxicity, the widespread use of non-viral carriers in clinical practice is still limited due to the insufficient efficacy associated with the difficulties of overcoming extracellular and intracellular barriers. Overcoming barriers by non-viral carriers is facilitated by their chemical structure, surface charge, as well as developed modifications. Currently, there are many different forms of non-viral carriers for various applications. This review aimed to summarize recent developments based on the essential requirements for non-viral carriers for gene therapy.
在过去几十年中,非病毒DNA和RNA递送系统作为病毒载体的替代物得到了深入研究。尽管相对于病毒具有最显著的优势,如缺乏免疫原性和细胞毒性,但由于与克服细胞外和细胞内屏障困难相关的疗效不足,非病毒载体在临床实践中的广泛应用仍然有限。非病毒载体的化学结构、表面电荷以及已开发的修饰有助于其克服屏障。目前,有许多不同形式的非病毒载体用于各种应用。本综述旨在根据基因治疗中非病毒载体的基本要求总结近期进展。