社论：“囊性纤维化治疗方法”特刊
Editorial: Special Issue on "Therapeutic Approaches for Cystic Fibrosis".
机构信息
UOC Genetica Medica, IRCCS Giannina Gaslini, 16147 Genova, Italy.
出版信息
Int J Mol Sci. 2020 Sep 11;21(18):6657. doi: 10.3390/ijms21186657.
Abstract
Cystic fibrosis (CF) is the most common lethal genetic disease in Caucasian populations, occurring in approximately 1 in 3000 newborns worldwide [...].
摘要
囊性纤维化（CF）是白种人群中最常见的致命性遗传疾病，全球约每 3000 名新生儿中就有 1 名患有该病[... ]。
相似文献
1
Editorial: Special Issue on "Therapeutic Approaches for Cystic Fibrosis".社论：“囊性纤维化治疗方法”特刊
Int J Mol Sci. 2020 Sep 11;21(18):6657. doi: 10.3390/ijms21186657.
2
Unique mutations of the cystic fibrosis transmembrane conductance regulator gene of three cases of cystic fibrosis in Nagasaki, Japan.日本长崎三例囊性纤维化患者囊性纤维化跨膜传导调节基因的独特突变
Intern Med. 2009;48(15):1327-31. doi: 10.2169/internalmedicine.48.2078. Epub 2009 Aug 3.
3
Pharmacogenetics of cystic fibrosis treatment.囊性纤维化治疗的药物遗传学
Pharmacogenomics. 2016 Aug;17(13):1453-63. doi: 10.2217/pgs.16.25. Epub 2016 Aug 4.
4
Distribution of CFTR mutations in cystic fibrosis patients of Tunisian origin: identification of two novel mutations.
Eur J Hum Genet. 1996;4(1):20-4. doi: 10.1159/000472165.
5
[Cystic fibrosis: relationship between genotype and phenotype].[囊性纤维化：基因型与表型之间的关系]
Arch Pediatr. 2003 Sep;10 Suppl 2:318s-324s. doi: 10.1016/s0929-693x(03)90046-7.
6
Clinical implications of cystic fibrosis transmembrane conductance regulator mutations.囊性纤维化跨膜传导调节因子突变的临床意义
Clin Chest Med. 1998 Sep;19(3):443-58, v. doi: 10.1016/s0272-5231(05)70092-7.
7
Cystic fibrosis transmembrane conductance regulator-mRNA delivery: a novel alternative for cystic fibrosis gene therapy.囊性纤维化跨膜电导调节因子-mRNA 递呈：囊性纤维化基因治疗的新选择。
J Gene Med. 2013 Nov-Dec;15(11-12):414-26. doi: 10.1002/jgm.2748.
8
[Modifier genes and cystic fibrosis].[修饰基因与囊性纤维化]
Arch Pediatr. 2006 Jan;13(1):57-63. doi: 10.1016/j.arcped.2005.09.029. Epub 2005 Nov 7.
9
Molecular Genetics of Cystic Fibrosis Transmembrane Conductance Regulator: Genotype and Phenotype.囊性纤维化跨膜传导调节因子的分子遗传学：基因型与表型
Pediatr Clin North Am. 2016 Aug;63(4):585-98. doi: 10.1016/j.pcl.2016.04.002.
10
Sweat chloride and immunoreactive trypsinogen in infants carrying two mutations and not affected by cystic fibrosis.携带两个突变且未受囊性纤维化影响的婴儿的汗液氯化物和免疫反应性胰蛋白酶原
Arch Dis Child. 2017 Jul;102(7):644-646. doi: 10.1136/archdischild-2015-309348. Epub 2016 Jan 11.
本文引用的文献
1
From Ivacaftor to Triple Combination: A Systematic Review of Efficacy and Safety of CFTR Modulators in People with Cystic Fibrosis.从 Ivacaftor 到三联组合：囊性纤维化患者 CFTR 调节剂疗效和安全性的系统评价。
Int J Mol Sci. 2020 Aug 16;21(16):5882. doi: 10.3390/ijms21165882.
2
Phosphorylation of the Chaperone-Like HspB5 Rescues Trafficking and Function of F508del-CFTR.磷酸化伴侣样热休克蛋白 B5 可恢复 F508del-CFTR 的运输和功能。
Int J Mol Sci. 2020 Jul 8;21(14):4844. doi: 10.3390/ijms21144844.
3
GM1 as Adjuvant of Innovative Therapies for Cystic Fibrosis Disease.GM1 作为囊性纤维化疾病创新疗法的佐剂。
Int J Mol Sci. 2020 Jun 24;21(12):4486. doi: 10.3390/ijms21124486.
4
The Effect of Sodium Bicarbonate, a Beneficial Adjuvant Molecule in Cystic Fibrosis, on Bronchial Epithelial Cells Expressing a Wild-Type or Mutant CFTR Channel.碳酸氢钠对表达野生型或突变 CFTR 通道的支气管上皮细胞的影响。囊性纤维化的有益辅助分子。
Int J Mol Sci. 2020 Jun 4;21(11):4024. doi: 10.3390/ijms21114024.
5
Gene Therapy for Cystic Fibrosis: Progress and Challenges of Genome Editing.囊性纤维化的基因治疗：基因组编辑的进展与挑战。
Int J Mol Sci. 2020 May 30;21(11):3903. doi: 10.3390/ijms21113903.
6
The Role of MicroRNA in the Airway Surface Liquid Homeostasis.微小 RNA 在气道表面液体动态平衡中的作用。
Int J Mol Sci. 2020 May 28;21(11):3848. doi: 10.3390/ijms21113848.
7
Synthesis and Therapeutic Applications of Iminosugars in Cystic Fibrosis.合成与应用在囊性纤维化治疗中的亚氨基糖
Int J Mol Sci. 2020 May 9;21(9):3353. doi: 10.3390/ijms21093353.
8
Immunomodulation in Cystic Fibrosis: Why and How?囊性纤维化的免疫调节：为何与如何？
Int J Mol Sci. 2020 May 8;21(9):3331. doi: 10.3390/ijms21093331.
9
What Role Does CFTR Play in Development, Differentiation, Regeneration and Cancer?囊性纤维化跨膜传导调节因子（CFTR）在发育、分化、再生和癌症中扮演什么角色？
Int J Mol Sci. 2020 Apr 29;21(9):3133. doi: 10.3390/ijms21093133.
10
Mechanical Properties of Human Bronchial Epithelial Cells Expressing Wt- and Mutant CFTR.人支气管上皮细胞中表达 wt 和突变 CFTR 的机械性能。
Int J Mol Sci. 2020 Apr 21;21(8):2916. doi: 10.3390/ijms21082916.
Zotero 插件