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American Association for the Advancement of Science, Science Advances, 8(6), 2020

DOI: 10.1126/sciadv.aay9669

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

Journal article published in 2020 by Nicoletta Pedemonte ORCID, Fabio Bertozzi ORCID, Emanuela Caci, Federico Sorana ORCID, Paolo Di Fruscia, Valeria Tomati, Loretta Ferrera, Alejandra Rodríguez-Gimeno ORCID, Francesco Berti ORCID, Emanuela Pesce, Elvira Sondo, Ambra Gianotti, Paolo Scudieri, Tiziano Bandiera ORCID, Luis J. V. Galietta ORCID
This paper is made freely available by the publisher.
This paper is made freely available by the publisher.

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Abstract

We identified a small molecule that, at picomolar concentrations, rescues mutant CFTR chloride channel from protein degradation.