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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Detalles Bibliográficos
Autores principales: Pibiri, Ivana, Melfi, Raffaella, Tutone, Marco, Di Leonardo, Aldo, Pace, Andrea, Lentini, Laura
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7504161/
https://www.ncbi.nlm.nih.gov/pubmed/32899265
http://dx.doi.org/10.3390/ijms21176420

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7504161/
https://www.ncbi.nlm.nih.gov/pubmed/32899265
http://dx.doi.org/10.3390/ijms21176420

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