Article

Suppression of nonsense mutations by small, cyclic peptides

2025-04-26

Abstract excerpt

<h4>ABSTRACT</h4> Premature termination codons in mRNAs result from nonsense mutations and hinder the translation of full-length, functional proteins. Nonsense mutations cause numerous serious genetic diseases, including cystic fibrosis and Duchenne muscular dystrophy. Several small-molecule drugs have been reported that could potentially ameliorate these diseases by promoting translational readthrough at the pre...

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Literature Corpus work: 30974142-146a-5edd-9aad-edb380a31faf
DOI: 10.1101/2025.04.25.650553

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