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Article

Targeting Nonsense: Optimization of 1,2,4-Oxadiazole TRIDs to Rescue CFTR Expression and Functionality in Cystic Fibrosis Cell Model Systems.

International journal of molecular sciences - 3 Sept 2020

Pibiri Ivana, Melfi Raffaella, Tutone Marco, Di Leonardo Aldo, Pace Andrea, Lentini Laura

Abstract excerpt

Cystic fibrosis (CF) patients develop a severe form of the disease when the cystic fibrosis transmembrane conductance regulator (CFTR) gene is affected by nonsense mutations. Nonsense mutations are responsible for the presence of a premature termination codon (PTC) in the mRNA, creating a lack of functional protein. In this context, translational readthrough-inducing drugs (TRIDs) represent a promising approach...

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Identifiers

PMID: 32899265
DOI: 10.3390/ijms21176420
PMCID: PMC7504161

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