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Drug-induced eRF1 degradation promotes readthrough and reveals a new branch of ribosome quality control

30 Aug 2023

Abstract excerpt

Suppression of premature termination codons (PTCs) by translational readthrough is a promising strategy to treat a wide variety of severe genetic diseases caused by nonsense mutations. Here, we present two potent readthrough promoters-NVS1.1 and NVS2.1-that restore substantial levels of functional full-length CFTR and IDUA proteins in disease models for cystic fibrosis and Hurler syndrome, respectively. In...

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PMID: 37651229
DOI: 10.1016/j.celrep.2023.113056

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