The arc of discovery, from the description of cystic fibrosis to effective treatments.
作者信息
Welsh Michael J
出版信息
J Clin Invest. 2024 Oct 1;134(19):e186231. doi: 10.1172/JCI186231.
Abstract
摘要
相似文献
1
The arc of discovery, from the description of cystic fibrosis to effective treatments.从囊性纤维化的描述到有效治疗方法的探索历程。
J Clin Invest. 2024 Oct 1;134(19):e186231. doi: 10.1172/JCI186231.
2
Human genetics: One gene, twenty years.人类遗传学：一个基因，二十年。
Nature. 2009 Jul 9;460(7252):164-9. doi: 10.1038/460164a.
3
Genetics. The promise of a cure: 20 years and counting.遗传学。治愈的希望：20年仍在继续。
Science. 2009 Jun 19;324(5934):1504-7. doi: 10.1126/science.324_1504.
4
A combination therapy for cystic fibrosis.一种用于囊性纤维化的联合疗法。
Cell. 2015 Sep 24;163(1):17. doi: 10.1016/j.cell.2015.09.003.
5
Genetic heterogeneity and cystic fibrosis.
Hum Mutat. 2009 Jul;30(7):v. doi: 10.1002/humu.21062.
6
From the farm to the lab: the pig as a new model of cystic fibrosis lung disease.从农场到实验室：猪作为囊性纤维化肺病的新模型
Am J Physiol Lung Cell Mol Physiol. 2008 Aug;295(2):L238-9. doi: 10.1152/ajplung.90311.2008. Epub 2008 May 23.
7
[Historical compilation of cystic fibrosis].
Gastroenterol Hepatol. 2016 Jan;39(1):36-42. doi: 10.1016/j.gastrohep.2015.04.012. Epub 2015 Jun 10.
8
The porcine lung as a potential model for cystic fibrosis.猪肺作为囊性纤维化的潜在模型。
Am J Physiol Lung Cell Mol Physiol. 2008 Aug;295(2):L240-63. doi: 10.1152/ajplung.90203.2008. Epub 2008 May 16.
9
Topical cystic fibrosis transmembrane conductance regulator gene replacement for cystic fibrosis-related lung disease.用于囊性纤维化相关肺部疾病的局部囊性纤维化跨膜传导调节因子基因替代疗法。
Paediatr Respir Rev. 2017 Mar;22:47-49. doi: 10.1016/j.prrv.2016.10.005. Epub 2016 Nov 5.
10
delta F508 in cystic fibrosis: willing but not able.囊性纤维化中的ΔF508：有心无力。
Arch Dis Child. 1997 Mar;76(3):278-82. doi: 10.1136/adc.76.3.278.
引用本文的文献
1
A Global Perspective on Transition Models for Pediatric to Adult Cystic Fibrosis Care: What Has Been Made So Far?儿童至成人囊性纤维化护理过渡模式的全球视角：目前已取得哪些进展？
J Clin Med. 2024 Dec 6;13(23):7428. doi: 10.3390/jcm13237428.
本文引用的文献
1
Potentiation of BKCa channels by cystic fibrosis transmembrane conductance regulator correctors VX-445 and VX-121.囊性纤维化跨膜电导调节因子校正剂 VX-445 和 VX-121 对 BKCa 通道的增强作用。
J Clin Invest. 2024 Jul 2;134(16):e176328. doi: 10.1172/JCI176328.
2
Arteriovenous metabolomics in pigs reveals CFTR regulation of metabolism in multiple organs.猪的动静脉代谢组学研究揭示了 CFTR 对多个器官代谢的调节作用。
J Clin Invest. 2024 May 14;134(13):e174500. doi: 10.1172/JCI174500.
3
Cystic Fibrosis.囊性纤维化
N Engl J Med. 2023 Nov 2;389(18):1693-1707. doi: 10.1056/NEJMra2216474.
4
Triple Therapy for Cystic Fibrosis -Gating and -Residual Function Genotypes.囊性纤维化-门控和-残留功能基因型的三联疗法。
N Engl J Med. 2021 Aug 26;385(9):815-825. doi: 10.1056/NEJMoa2100665.
5
Origins of cystic fibrosis lung disease.囊性纤维化肺病的起源。
N Engl J Med. 2015 Jan 22;372(4):351-62. doi: 10.1056/NEJMra1300109.
6
Transgenic animals may help resolve a sticky situation in cystic fibrosis.转基因动物可能有助于解决囊性纤维化中的一个棘手问题。
J Clin Invest. 2010 Sep;120(9):3093-6. doi: 10.1172/JCI44235. Epub 2010 Aug 25.
7
A molecular abnormality of urinary mucoprotein in cystic fibrosis of the pancreas.胰腺囊性纤维化中尿粘蛋白的分子异常。
J Clin Invest. 1962 Mar;41(3):455-62. doi: 10.1172/JCI104498.
8
9
Airway epithelial CFTR mRNA expression in cystic fibrosis patients after repetitive administration of a recombinant adenovirus.重复给予重组腺病毒后囊性纤维化患者气道上皮CFTR mRNA表达情况
J Clin Invest. 1999 Nov;104(9):1245-55. doi: 10.1172/JCI7935.
10
Comparison of DNA-lipid complexes and DNA alone for gene transfer to cystic fibrosis airway epithelia in vivo.体内将DNA-脂质复合物与单独的DNA用于基因转移至囊性纤维化气道上皮细胞的比较。
J Clin Invest. 1997 Sep 15;100(6):1529-37. doi: 10.1172/JCI119676.
Zotero 插件