ArticlePloS one2014
Correcting the cystic fibrosis disease mutant, A455E CFTR.
Article in PloS one, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
10 citing papers in PubMed.
- In vivo and in vitro ivacaftor response in cystic fibrosis patients with residual CFTR function: N-of-1 studies.Pediatric pulmonology · 2017Trial
- Cystic Fibrosis Human Organs-on-a-Chip.Micromachines · 2021Review
- Phenotyping of RareCells · 2020Article
- Steric Inhibition of 5' UTR Regulatory Elements Results in Upregulation of Human CFTR.Molecular therapy : the journal of the American Society of Gene Therapy · 2019Article
- Review
- Rescue of CFTR NBD2 mutants N1303K and S1235R is influenced by the functioning of the autophagosome.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2018Article
- Barriers to inhaled gene therapy of obstructive lung diseases: A review.Journal of controlled release : official journal of the Controlled Release Society · 2016Review
- Correctors Rescue CFTR Mutations in Nucleotide-Binding Domain 1 (NBD1) by Modulating Proteostasis.Chembiochem : a European journal of chemical biology · 2016Article
- Combination of Correctors Rescue ΔF508-CFTR by Reducing Its Association with Hsp40 and Hsp27.The Journal of biological chemistry · 2015Article
- Rescue of NBD2 mutants N1303K and S1235R of CFTR by small-molecule correctors and transcomplementation.PloS one · 2015Article
Corrections and comments
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Authors and funding
4 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Cystic fibrosis is caused by more than 1000 mutations, the most common being the ΔF508 mutation. These mutations have been divided into five classes [1], with ΔF508 CFTR in class II. Here we have studied the class V mutation A455E. We report that the mature and immature bands of A455E are rapidly degraded primarily by proteasomes; the short protein half-life of this mutant therefore resembles that of ΔF508 CFTR. A455E could be rescued by treatment of the cells with proteasome inhibitors. Furthermore, co-transfection of A455E with the truncation mutant Δ264 CFTR also rescued the mature C band, indicating that A455E can be rescued by transcomplementation. We found that Δ264 CFTR bound to A455E, forming a bimolecular complex. Treatment with the compound correctors C3 and C4 also rescued A455E. These results are significant because they show that although ΔF508 belongs to a different class than A455E, it can be rescued by the same strategies, offering therapeutic promise to patients with Class V mutations.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.