Discovery of a picomolar potency pharmacological corrector of the mutant CFTR chloride channel

F508del, the most frequent mutation causing cystic fibrosis (CF), results in mistrafficking and premature degradation of the CFTR chloride channel. Small molecules named correctors may rescue F508del-CFTR and therefore represent promising drugs to target the basic defect in CF. We screened a careful...

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Detalles Bibliográficos
Autores principales: Pedemonte, Nicoletta, Bertozzi, Fabio, Caci, Emanuela, Sorana, Federico, Di Fruscia, Paolo, Tomati, Valeria, Ferrera, Loretta, Rodríguez-Gimeno, Alejandra, Berti, Francesco, Pesce, Emanuela, Sondo, Elvira, Gianotti, Ambra, Scudieri, Paolo, Bandiera, Tiziano, Galietta, Luis J. V.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Association for the Advancement of Science 2020
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7034990/
https://www.ncbi.nlm.nih.gov/pubmed/32128418
http://dx.doi.org/10.1126/sciadv.aay9669

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