Article

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

2023-01-31

Abstract excerpt

<h4>Summary</h4> Suppression of premature termination codons (PTC) by translational readthrough is a promising strategy to treat a wide variety of severe genetic diseases caused by nonsense mutations. Here, we present two novel and 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 syn...

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Literature Corpus work: 545026b2-6350-502b-bf96-3cc55c7f948d
DOI: 10.1101/2023.01.31.526456

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