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
The era of CFTR modulators: improvements made and remaining challenges.
作者信息
Cuevas-Ocaña Sara, Laselva Onofrio, Avolio Julie, Nenna Raffaella
机构信息
Wolfson Centre for Stem Cells, Tissue Engineering and Modelling, School of Medicine, University of Nottingham Biodiscovery Institute, Nottingham, UK.
Program in Molecular Medicine, The Hospital for Sick Children, Toronto, Canada.
出版信息
Breathe (Sheff). 2020 Jun;16(2):200016. doi: 10.1183/20734735.0016-2020.
Abstract
摘要
https://bit.ly/3aRafQF. （此为网址，直接保留原文即可，无法准确翻译为中文，若强行翻译为“https://短链接/3aRafQF”，不符合网址翻译要求，故保留原文）
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本文引用的文献
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Biomolecules. 2020 Feb 19;10(2):334. doi: 10.3390/biom10020334.
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Adult Care in Cystic Fibrosis.囊性纤维化成人护理。
Semin Respir Crit Care Med. 2019 Dec;40(6):857-868. doi: 10.1055/s-0039-3400289. Epub 2019 Dec 30.
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The influence of CFTR complex alleles on precision therapy of cystic fibrosis.CFTR 复合等位基因对囊性纤维化精准治疗的影响。
J Cyst Fibros. 2020 Mar;19 Suppl 1:S15-S18. doi: 10.1016/j.jcf.2019.12.008. Epub 2019 Dec 26.
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The genetics and genomics of cystic fibrosis.囊性纤维化的遗传学和基因组学。
J Cyst Fibros. 2020 Mar;19 Suppl 1(Suppl 1):S5-S9. doi: 10.1016/j.jcf.2019.11.003. Epub 2019 Dec 23.
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Utilizing centralized biorepository samples for biomarkers of cystic fibrosis lung disease severity.利用集中的生物库样本进行囊性纤维化肺病严重程度的生物标志物研究。
J Cyst Fibros. 2020 Jul;19(4):632-640. doi: 10.1016/j.jcf.2019.12.007. Epub 2019 Dec 20.
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Lumacaftor/ivacaftor reduces exacerbations in adults homozygous for Phe508del mutation with severe lung disease.Lumacaftor/ivacaftor 可减少严重肺部疾病纯合子 Phe508del 突变患者的恶化次数。
J Cyst Fibros. 2020 May;19(3):415-420. doi: 10.1016/j.jcf.2019.12.006. Epub 2019 Dec 15.
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Functional rescue of c.3846G>A (W1282X) in patient-derived nasal cultures achieved by inhibition of nonsense mediated decay and protein modulators with complementary mechanisms of action.通过抑制无意义介导的衰变和具有互补作用机制的蛋白质调节剂，在患者来源的鼻培养物中实现了 c.3846G>A(W1282X) 的功能挽救。
J Cyst Fibros. 2020 Sep;19(5):717-727. doi: 10.1016/j.jcf.2019.12.001. Epub 2019 Dec 9.
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Elexacaftor/Ivacaftor/Tezacaftor: First Approval.依伐卡托/埃乐卡福特/泰泽卡福特：首次批准。
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