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Article

Toward a rationale for the PTC124 (Ataluren) promoted readthrough of premature stop codons: a computational approach and GFP-reporter cell-based assay.

Molecular pharmaceutics - 3 Mar 2014

Lentini Laura, Melfi Raffaella, Di Leonardo Aldo, Spinello Angelo, Barone Giampaolo, Pace Andrea, Palumbo Piccionello Antonio, Pibiri Ivana

Abstract excerpt

The presence in the mRNA of premature stop codons (PTCs) results in protein truncation responsible for several inherited (genetic) diseases. A well-known example of these diseases is cystic fibrosis (CF), where approximately 10% (worldwide) of patients have nonsense mutations in the CF transmembrane regulator (CFTR) gene. PTC124 (3-(5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl)-benzoic acid), also known as Ataluren,...

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Source

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Identifiers

PMID: 24483936
DOI: 10.1021/mp400230s
PMCID: PMC4167060

Genes

CFTR

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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