Suppressor tRNA in gene therapy  

作　　者：Jingjing Ruan Xiaoxiao Yu Huixia Xu Wenrui Cui Kaiye Zhang Chenyang Liu Wenlong Sun Xiaodan Huang Lei An Yue Zhang 

机构地区：[1]The Children's Hospital,National Clinical Research Center for Child Health,Zhejiang University School of Medicine,Liangzhu Laboratory,Hangzhou 310000,China [2]Liangzhu Laboratory,Zhejiang University School of Medicine,Hangzhou 311121,China [3]Department of Thoracic and Cardiovascular Surgery,Huaihe Hospital of Henan University,Henan University,Kaifeng 475000,China [4]Translational Medicine Center,Huaihe Hospital of Henan University,Henan University,Kaifeng 475000,China [5]Eye Center,The Second Affiliated Hospital,School of Medicine,Zhejiang University,Hangzhou 310009,China

出　　处：《Science China(Life Sciences)》2024年第10期2120-2131,共12页中国科学（生命科学英文版）

基　　金：supported by the National Natural Science Foundation of China(82371861);Key R&D Program of Zhejiang(2024SSYS0020);Henan Province Key Research and Promotion Project(242102311023);the Starting Fund from Zhejiang University。

摘　　要：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 t RNAs 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 t RNAs,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.

关 键 词：suppressor tRNA PTC nonsense mutation READTHROUGH gene therapy 

分 类 号：R450[医药卫生—治疗学]