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Comprehensive Analysis of Combinatorial Pharmacological Treatments to Correct Nonsense Mutations in the CFTR Gene

Cystic fibrosis (CF) is caused by loss of function of the CFTR chloride channel. A substantial number of CF patients carry nonsense mutations in the CFTR gene. These patients cannot directly benefit from pharmacological correctors and potentiators that have been developed for other types of CFTR mut...

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Detalles Bibliográficos
Autores principales: Venturini, Arianna, Borrelli, Anna, Musante, Ilaria, Scudieri, Paolo, Capurro, Valeria, Renda, Mario, Pedemonte, Nicoletta, Galietta, Luis J. V.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8584557/
https://www.ncbi.nlm.nih.gov/pubmed/34769402
http://dx.doi.org/10.3390/ijms222111972

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