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Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Ubiquitylation as a Novel Pharmaceutical Target for Cystic Fibrosis

Department of Biomedical Chemistry, School of Science and Technology, Kwansei Gakuin University, 2-1 Gakuen, Sanda, Hyogo 669-1337, Japan
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Pharmaceuticals 2020, 13(4), 75; https://doi.org/10.3390/ph13040075
Received: 3 March 2020 / Revised: 20 April 2020 / Accepted: 20 April 2020 / Published: 22 April 2020
(This article belongs to the Special Issue Targeted Protein Degradation: From Chemical Biology to Drug Discovery)
Mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene decrease the structural stability and function of the CFTR protein, resulting in cystic fibrosis. Recently, the effect of CFTR-targeting combination therapy has dramatically increased, and it is expected that add-on drugs that modulate the CFTR surrounding environment will further enhance their effectiveness. Various interacting proteins have been implicated in the structural stability of CFTR and, among them, molecules involved in CFTR ubiquitylation are promising therapeutic targets as regulators of CFTR degradation. This review focuses on the ubiquitylation mechanism that contributes to the stability of mutant CFTR at the endoplasmic reticulum (ER) and post-ER compartments and discusses the possibility as a pharmacological target for cystic fibrosis (CF). View Full-Text
Keywords: cystic fibrosis transmembrane conductance regulator (CFTR); endoplasmic reticulum quality control (ERQC); plasma membrane quality control (PMQC); ubiquitylation; chaperone; cystic fibrosis (CF) treatment cystic fibrosis transmembrane conductance regulator (CFTR); endoplasmic reticulum quality control (ERQC); plasma membrane quality control (PMQC); ubiquitylation; chaperone; cystic fibrosis (CF) treatment
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MDPI and ACS Style

Fukuda, R.; Okiyoneda, T. Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Ubiquitylation as a Novel Pharmaceutical Target for Cystic Fibrosis. Pharmaceuticals 2020, 13, 75. https://doi.org/10.3390/ph13040075

AMA Style

Fukuda R, Okiyoneda T. Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Ubiquitylation as a Novel Pharmaceutical Target for Cystic Fibrosis. Pharmaceuticals. 2020; 13(4):75. https://doi.org/10.3390/ph13040075

Chicago/Turabian Style

Fukuda, Ryosuke, and Tsukasa Okiyoneda. 2020. "Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Ubiquitylation as a Novel Pharmaceutical Target for Cystic Fibrosis" Pharmaceuticals 13, no. 4: 75. https://doi.org/10.3390/ph13040075

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