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Article

Enhancement of premature stop codon readthrough in the CFTR gene by Ataluren (PTC124) derivatives.

European journal of medicinal chemistry - 28 Aug 2015

Pibiri Ivana, Lentini Laura, Melfi Raffaella, Gallucci Giulia, Pace Andrea, Spinello Angelo, Barone Giampaolo, Di Leonardo Aldo

Abstract excerpt

Premature stop codons are the result of nonsense mutations occurring within the coding sequence of a gene. These mutations lead to the synthesis of a truncated protein and are responsible for several genetic diseases. A potential pharmacological approach to treat these diseases is to promote the translational readthrough of premature stop codons by small molecules aiming to restore the full-length protein. The...

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Source

Identifiers

PMID: 26142488
DOI: 10.1016/j.ejmech.2015.06.038

Genes

CFTR

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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