NVS2.1 is a potent, orally active and brain-penetrant readthrough promoter. NVS2.1 triggers the degradation of eRF1 by activating the ribosome-related quality control pathways (involving GCN1, RNF14, and RNF25), thereby facilitating the reading through of premature termination codons. NVS2.1 can be used for the research of genetic diseases caused by nonsense mutations.
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