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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Detalhes bibliográficos
Publicado no:Sci Adv
Main Authors: 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
Idioma:Inglês
Publicado em: American Association for the Advancement of Science 2020
Assuntos:
Research Articles
Acesso em linha: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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