Suppressor tRNA in gene therapy.

Autor: Ruan J; The Children's Hospital, National Clinical Research Center for Child Health, Zhejiang University School of Medicine, Liangzhu Laboratory, Hangzhou, 310000, China.; Liangzhu Laboratory, Zhejiang University School of Medicine, Hangzhou, 311121, China., Yu X; Liangzhu Laboratory, Zhejiang University School of Medicine, Hangzhou, 311121, China., Xu H; Department of Thoracic and Cardiovascular Surgery, Huaihe Hospital of Henan University, Henan University, Kaifeng, 475000, China., Cui W; Translational Medicine Center, Huaihe Hospital of Henan University, Henan University, Kaifeng, 475000, China., Zhang K; Eye Center, The Second Affiliated Hospital, School of Medicine, Zhejiang University, Hangzhou, 310009, China., Liu C; Translational Medicine Center, Huaihe Hospital of Henan University, Henan University, Kaifeng, 475000, China., Sun W; Liangzhu Laboratory, Zhejiang University School of Medicine, Hangzhou, 311121, China., Huang X; Eye Center, The Second Affiliated Hospital, School of Medicine, Zhejiang University, Hangzhou, 310009, China., An L; Translational Medicine Center, Huaihe Hospital of Henan University, Henan University, Kaifeng, 475000, China. anlei@henu.edu.cn., Zhang Y; The Children's Hospital, National Clinical Research Center for Child Health, Zhejiang University School of Medicine, Liangzhu Laboratory, Hangzhou, 310000, China. yue.zhang@zju.edu.cn.; Liangzhu Laboratory, Zhejiang University School of Medicine, Hangzhou, 311121, China. yue.zhang@zju.edu.cn.; Translational Medicine Center, Huaihe Hospital of Henan University, Henan University, Kaifeng, 475000, China. yue.zhang@zju.edu.cn.
Jazyk: angličtina
Zdroj: Science China. Life sciences [Sci China Life Sci] 2024 Oct; Vol. 67 (10), pp. 2120-2131. Date of Electronic Publication: 2024 Jun 24.
DOI: 10.1007/s11427-024-2613-y
Abstrakt: 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.
(© 2024. Science China Press.)
Databáze: MEDLINE
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