Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Ubiquitylation as a Novel Pharmaceutical Target for Cystic Fibrosis
Mutations in the cystic fibrosis transmembrane conductance regulator (<i>CFTR)</i> 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 expec...
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| Format: | Article |
| Language: | English |
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MDPI AG
2020-04-01
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| Series: | Pharmaceuticals |
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| Online Access: | https://www.mdpi.com/1424-8247/13/4/75 |
| _version_ | 1797569821159718912 |
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| author | Ryosuke Fukuda Tsukasa Okiyoneda |
| author_facet | Ryosuke Fukuda Tsukasa Okiyoneda |
| author_sort | Ryosuke Fukuda |
| collection | DOAJ |
| description | Mutations in the cystic fibrosis transmembrane conductance regulator (<i>CFTR)</i> 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). |
| first_indexed | 2024-03-10T20:17:00Z |
| format | Article |
| id | doaj.art-0319d70b1e3b4791898084c7df6cc1ec |
| institution | Directory Open Access Journal |
| issn | 1424-8247 |
| language | English |
| last_indexed | 2024-03-10T20:17:00Z |
| publishDate | 2020-04-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Pharmaceuticals |
| spelling | doaj.art-0319d70b1e3b4791898084c7df6cc1ec2023-11-19T22:25:59ZengMDPI AGPharmaceuticals1424-82472020-04-011347510.3390/ph13040075Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Ubiquitylation as a Novel Pharmaceutical Target for Cystic FibrosisRyosuke Fukuda0Tsukasa Okiyoneda1Department of Biomedical Chemistry, School of Science and Technology, Kwansei Gakuin University, 2-1 Gakuen, Sanda, Hyogo 669-1337, JapanDepartment of Biomedical Chemistry, School of Science and Technology, Kwansei Gakuin University, 2-1 Gakuen, Sanda, Hyogo 669-1337, JapanMutations in the cystic fibrosis transmembrane conductance regulator (<i>CFTR)</i> 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).https://www.mdpi.com/1424-8247/13/4/75cystic fibrosis transmembrane conductance regulator (CFTR)endoplasmic reticulum quality control (ERQC)plasma membrane quality control (PMQC)ubiquitylationchaperonecystic fibrosis (CF) treatment |
| spellingShingle | Ryosuke Fukuda Tsukasa Okiyoneda Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Ubiquitylation as a Novel Pharmaceutical Target for Cystic Fibrosis Pharmaceuticals cystic fibrosis transmembrane conductance regulator (CFTR) endoplasmic reticulum quality control (ERQC) plasma membrane quality control (PMQC) ubiquitylation chaperone cystic fibrosis (CF) treatment |
| title | Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Ubiquitylation as a Novel Pharmaceutical Target for Cystic Fibrosis |
| title_full | Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Ubiquitylation as a Novel Pharmaceutical Target for Cystic Fibrosis |
| title_fullStr | Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Ubiquitylation as a Novel Pharmaceutical Target for Cystic Fibrosis |
| title_full_unstemmed | Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Ubiquitylation as a Novel Pharmaceutical Target for Cystic Fibrosis |
| title_short | Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Ubiquitylation as a Novel Pharmaceutical Target for Cystic Fibrosis |
| title_sort | cystic fibrosis transmembrane conductance regulator cftr ubiquitylation as a novel pharmaceutical target for cystic fibrosis |
| topic | cystic fibrosis transmembrane conductance regulator (CFTR) endoplasmic reticulum quality control (ERQC) plasma membrane quality control (PMQC) ubiquitylation chaperone cystic fibrosis (CF) treatment |
| url | https://www.mdpi.com/1424-8247/13/4/75 |
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