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Read Through Of Truncated Proteins In Premature Termination Codon Diseases Using An Optimized Genetic Codon Expansion System
Read Through Of Truncated Proteins In Premature Termination Codon Diseases Using An Optimized Genetic Codon Expansion System
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机译:使用优化的遗传密码子扩展系统通读过早终止密码子疾病中的截短蛋白
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摘要
Provided is a method for high-efficiently reading through a nonsense mutation site in a pathogenic gene in a monogenic hereditary disease and restoring the normal structure and function of a mutant protein, by using a genetic code expanded non-natural amino acid system. By modifying a tRNA of Methanosarcina barkeri (tRNAPyl), an all-new UAA and UGA encoded non-natural amino acid system that has high read-through efficiency is obtained, and the range of using the orthogonal pair of tRNAPyl and pyrrolysyl-tRNA synthetase (PylRS) is expanded. A plasmid mimicking the endogenous premature termination codon is constructed, so as to evaluate the efficiency of reading through the endogenous premature termination codon. Also provided is a system mainly comprising pathogenic genes of monogenic hereditary diseases and tumor inhibitory genes in tumor cells.
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