Article

Genome-scale quantification and prediction of pathogenic stop codon readthrough by small molecules

2023-08-08

Abstract excerpt

Premature termination codons (PTCs) cause ∼10-20% of Mendelian diseases and are the major mechanism of tumor suppressor gene inactivation in cancer. A general strategy to alleviate the effects of PTCs would be to promote translational readthrough. Nonsense suppression by small molecules has proven effective in diverse disease models, but translation into the clinic is hampered by ineffective readthrough of many PT...

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Literature Corpus work: 8c266eb2-bea8-5d32-85d9-f231d3c129f9
DOI: 10.1101/2023.08.07.552350

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