Article
CRISPR-free, programmable RNA pseudouridylation to suppress premature termination codons.
Molecular cell - 5 Jan 2023
Song Jinghui, Dong Liting, Sun Hanxiao, Luo Nan, Huang Qiang, Li Kai, Shen Xiaowen, Jiang Zhe, Lv Zhicong, Peng Luxin, Zhang Meifang, Wang Kun, Liu Ke, Hong Jiaxu, Yi Chengqi
Abstract excerpt
Nonsense mutations, accounting for >20% of disease-associated mutations, lead to premature translation termination. Replacing uridine with pseudouridine in stop codons suppresses translation termination, which could be harnessed to mediate readthrough of premature termination codons (PTCs). Here, we present RESTART, a programmable RNA base editor, to revert PTC-induced translation termination in mammalian cells....
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