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Highly Efficient Gene Editing of Cystic Fibrosis Patient-Derived Airway Basal Cells Results in Functional CFTR Correction

scientific article published on 29 April 2020

Residual function of cystic fibrosis mutants predicts response to small molecule CFTR modulators

scientific article published on 26 July 2018

Restoration of W1282X CFTR activity by enhanced expression

artículo científico publicado en 2007

Robust Stimulation of W1282X-CFTR Channel Activity by a Combination of Allosteric Modulators

artículo científico publicado en 2016

Slowing ribosome velocity restores folding and function of mutant CFTR

scientific article published on 01 December 2019

The silent codon change I507-ATC->ATT contributes to the severity of the ΔF508 CFTR channel dysfunction

artículo científico publicado en 2013

The unfolded protein response (UPR)-activated transcription factor X-box-binding protein 1 (XBP1) induces microRNA-346 expression that targets the human antigen peptide transporter 1 (TAP1) mRNA and governs immune regulatory genes.

artículo científico publicado en 2011

VX-770-mediated potentiation of numerous human CFTR disease mutants is influenced by phosphorylation level

artículo científico publicado en 2019

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