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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Publicado en:Sci Adv
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: Artigo
Lenguaje:Inglês
Publicado: American Association for the Advancement of Science 2020
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Research Articles
Acceso en línea: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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