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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Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:Sci Adv
Prif Awduron: 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.
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: American Association for the Advancement of Science 2020
Pynciau:
Research Articles
Mynediad Ar-lein:https://ncbi.nlm.nih.gov/pmc/articles/PMC7034990/
https://ncbi.nlm.nih.gov/pubmed/32128418
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/sciadv.aay9669
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