基因治疗中的抑制性 tRNA。

IF 8 2区 生物学 Q1 BIOLOGY
Science China Life Sciences Pub Date : 2024-10-01 Epub Date: 2024-06-24 DOI:10.1007/s11427-024-2613-y
Jingjing Ruan, Xiaoxiao Yu, Huixia Xu, Wenrui Cui, Kaiye Zhang, Chenyang Liu, Wenlong Sun, Xiaodan Huang, Lei An, Yue Zhang
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引用次数: 0

摘要

无义突变导致编码序列中出现过早终止密码子(PTC),从而导致蛋白质被截断,通常是无功能蛋白质。抑制性 tRNA 可以识别这些 PTC 并与之配对,使核糖体继续翻译并产生全长蛋白质。这篇综述介绍了抑制性 tRNA 的机制和发展,比较了抑制性 tRNA 和其他读通疗法,讨论了它们在临床治疗中的潜力、局限性和障碍。我们还总结了抑制性 tRNA 在体外和体内的应用,为无义突变疾病的研究和治疗提供了新的见解。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Suppressor tRNA in gene therapy.

Suppressor tRNAs are engineered or naturally occurring transfer RNA molecules that have shown promise in gene therapy for diseases caused by nonsense mutations, which result in premature termination codons (PTCs) in coding sequence, leading to truncated, often nonfunctional proteins. Suppressor tRNAs can recognize and pair with these PTCs, allowing the ribosome to continue translation and produce a full-length protein. This review introduces the mechanism and development of suppressor tRNAs, compares suppressor tRNAs with other readthrough therapies, discusses their potential for clinical therapy, limitations, and obstacles. We also summarize the applications of suppressor tRNAs in both in vitro and in vivo, offering new insights into the research and treatment of nonsense mutation diseases.

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来源期刊
CiteScore
15.10
自引率
8.80%
发文量
2907
审稿时长
3.2 months
期刊介绍: Science China Life Sciences is a scholarly journal co-sponsored by the Chinese Academy of Sciences and the National Natural Science Foundation of China, and it is published by Science China Press. The journal is dedicated to publishing high-quality, original research findings in both basic and applied life science research.
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