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

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

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 fu...

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Bibliographic Details
Main Authors: Ivana Pibiri, Raffaella Melfi, Marco Tutone, Aldo Di Leonardo, Andrea Pace, Laura Lentini
Format: Article
Language:English
Published: MDPI AG 2020-09-01
Series:International Journal of Molecular Sciences
Subjects:
premature termination codon
nonsense mutation
genetic disorder
translational readthrough inducing drugs
oxadiazoles
Online Access:https://www.mdpi.com/1422-0067/21/17/6420

Internet

https://www.mdpi.com/1422-0067/21/17/6420

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