Read through of truncated proteins in premature termination codon diseases using an optimized genetic codon expansion system
US11597931B2 · kind B2 · utility
Assignee
Inventors
Key dates
| Filing date | Aug 6, 2020 |
| Grant date | Mar 7, 2023 |
| Priority date | — |
| Expiry date | Oct 6, 2040 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2310/335
- WIPO fieldMeasurement
- WIPO sectorInstruments
Abstract
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.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.