Editorial: Mechanisms of Novel Drugs and Gene Modifiers in the Treatment of Cystic Fibrosis.
作者信息
Veit Guido, Silva Iris, Conese Massimo, Laselva Onofrio
机构信息
Department of Physiology, McGill University, Montréal, QC, Canada.
S2AQUAcoLAB, Olhao, Portugal.
出版信息
Front Mol Biosci. 2022 Jul 12;9:975946. doi: 10.3389/fmolb.2022.975946. eCollection 2022.
Abstract
摘要
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本文引用的文献
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Gene-specific nonsense-mediated mRNA decay targeting for cystic fibrosis therapy.针对囊性纤维化治疗的基因特异性无意义介导的 mRNA 衰减。
Nat Commun. 2022 May 27;13(1):2978. doi: 10.1038/s41467-022-30668-y.
2
Antisense oligonucleotide splicing modulation as a novel Cystic Fibrosis therapeutic approach for the W1282X nonsense mutation.反义寡核苷酸剪接调节作为一种新型囊性纤维化治疗方法，针对 W1282X 无义突变。
J Cyst Fibros. 2022 Jul;21(4):630-636. doi: 10.1016/j.jcf.2021.12.012. Epub 2021 Dec 28.
3
Small-molecule drugs for cystic fibrosis: Where are we now?用于治疗囊性纤维化的小分子药物：我们目前处于什么阶段？
Pulm Pharmacol Ther. 2022 Feb;72:102098. doi: 10.1016/j.pupt.2021.102098. Epub 2021 Nov 15.
4
Comprehensive Analysis of Combinatorial Pharmacological Treatments to Correct Nonsense Mutations in the CFTR Gene.综合分析组合药理学治疗纠正 CFTR 基因无义突变。
Int J Mol Sci. 2021 Nov 4;22(21):11972. doi: 10.3390/ijms222111972.
5
The era of CFTR modulators: improvements made and remaining challenges.囊性纤维化跨膜传导调节因子调节剂时代：已取得的进展及尚存的挑战
Breathe (Sheff). 2020 Jun;16(2):200016. doi: 10.1183/20734735.0016-2020.
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A helper-dependent adenoviral vector rescues CFTR to wild-type functional levels in cystic fibrosis epithelial cells harbouring class I mutations.一种依赖辅助病毒的腺病毒载体可将囊性纤维化上皮细胞中携带I类突变的CFTR拯救至野生型功能水平。
Eur Respir J. 2020 Nov 12;56(5). doi: 10.1183/13993003.00205-2020. Print 2020 Nov.
7
Prolonged co-treatment with HGF sustains epithelial integrity and improves pharmacological rescue of Phe508del-CFTR.持续的 HGF 共处理维持上皮完整性，并改善 Phe508del-CFTR 的药物解救。
Sci Rep. 2018 Aug 29;8(1):13026. doi: 10.1038/s41598-018-31514-2.
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Chaperone-Independent Peripheral Quality Control of CFTR by RFFL E3 Ligase.RFFL E3 连接酶对 CFTR 的无伴侣蛋白的周围质量控制。
Dev Cell. 2018 Mar 26;44(6):694-708.e7. doi: 10.1016/j.devcel.2018.02.001. Epub 2018 Mar 1.
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Mol Biol Cell. 2016 Feb 1;27(3):424-33. doi: 10.1091/mbc.E14-04-0935.
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Sci Transl Med. 2014 Jul 23;6(246):246ra97. doi: 10.1126/scitranslmed.3008889.
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