少有人走的路：英国呼吸基因治疗联盟在囊性纤维化基因治疗方面稳步前行 - Suppr

Suppr

超能文献

The Road Less Traveled: Slow but Steady Progress Toward Cystic Fibrosis Gene Therapy by the UK Respiratory Gene Therapy Consortium.

作者信息

Flotte Terence R

机构信息

Horae Gene Therapy Center University of Massachusetts Chan Medical School Worcester, Massachusetts.

出版信息

Am J Respir Crit Care Med. 2024 Dec 15;210(12):1387-1389. doi: 10.1164/rccm.202408-1516ED.

DOI:10.1164/rccm.202408-1516ED

PMID:39265185

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11716029/

Abstract

摘要

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/adde/11716029/44de708f7de1/rccm.202408-1516EDf1.jpg

相似文献

The Road Less Traveled: Slow but Steady Progress Toward Cystic Fibrosis Gene Therapy by the UK Respiratory Gene Therapy Consortium.少有人走的路：英国呼吸基因治疗联盟在囊性纤维化基因治疗方面稳步前行

Am J Respir Crit Care Med. 2024 Dec 15;210(12):1387-1389. doi: 10.1164/rccm.202408-1516ED.

Gene therapy in cystic fibrosis.囊性纤维化的基因治疗。

Arch Dis Child. 2014 May;99(5):465-8. doi: 10.1136/archdischild-2012-302158. Epub 2014 Jan 24.

Perceived burden of respiratory physiotherapy in people with cystic fibrosis taking elexacaftor-tezacaftor-ivacaftor combination: a 1-year observational study.囊性纤维化患者使用依利卓艾卡托替卡那三联组合治疗的呼吸物理治疗感知负担：一项为期 1 年的观察性研究。

Ther Adv Respir Dis. 2024 Jan-Dec;18:17534666241235054. doi: 10.1177/17534666241235054.

A randomised, double-blind, placebo-controlled phase IIB clinical trial of repeated application of gene therapy in patients with cystic fibrosis.一项随机、双盲、安慰剂对照的 IIB 期临床试验，评估在囊性纤维化患者中重复应用基因治疗的效果。

Thorax. 2013 Nov;68(11):1075-7. doi: 10.1136/thoraxjnl-2013-203309. Epub 2013 Mar 22.

Cystic Fibrosis Gene Therapy in the UK and Elsewhere.英国及其他地区的囊性纤维化基因治疗

Hum Gene Ther. 2015 May;26(5):266-75. doi: 10.1089/hum.2015.027.

Human Molecular Genetics and the long road to treating cystic fibrosis.人类分子遗传学与囊性纤维化治疗的漫漫征途。

Hum Mol Genet. 2021 Oct 1;30(R2):R264-R273. doi: 10.1093/hmg/ddab191.

Noninvasive Ventilation in Cystic Fibrosis: Clinical Indications and Outcomes in a Large UK Adult Cystic Fibrosis Center.囊性纤维化中的无创通气：大型英国成人囊性纤维化中心的临床适应证和结局。

Respir Care. 2021 Mar;66(3):466-474. doi: 10.4187/respcare.07862. Epub 2020 Sep 8.

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。

Cochrane Database Syst Rev. 2016 Jun 17;2016(6):CD005599. doi: 10.1002/14651858.CD005599.pub5.

Gene therapy for cystic fibrosis: steady progress, should do well.囊性纤维化的基因治疗：进展稳健，前景良好。

Eur Respir J. 1997 Feb;10(2):257-9. doi: 10.1183/09031936.97.10020257.

Progress in gene and cell therapy for cystic fibrosis lung disease.囊性纤维化肺病的基因和细胞治疗进展。

Curr Pharm Des. 2012;18(5):642-62. doi: 10.2174/138161212799315993.

引用本文的文献

Progress of personalized medicine of cystic fibrosis in the times of efficient CFTR modulators.高效CFTR调节剂时代囊性纤维化个性化医疗的进展

Mol Cell Pediatr. 2025 May 5;12(1):6. doi: 10.1186/s40348-025-00194-0.

本文引用的文献

Lentiviral Gene Therapy for Cystic Fibrosis: A Promising Approach and First-in-Human Trial.用于囊性纤维化的慢病毒基因疗法：一种有前景的方法及首次人体试验。

Am J Respir Crit Care Med. 2024 Dec 15;210(12):1398-1408. doi: 10.1164/rccm.202402-0389CI.

A Paradox of the Field's Own Success: Unintended Challenges in Bringing Cutting-Edge Science from the Bench to the Market.该领域自身成功带来的悖论：将前沿科学从实验室推向市场过程中意想不到的挑战。

Hum Gene Ther. 2024 Feb;35(3-4):83-88. doi: 10.1089/hum.2023.29264.nyi.

Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis.针对囊性纤维化患者的首次人体慢病毒试验的准备工作。

Thorax. 2017 Feb;72(2):137-147. doi: 10.1136/thoraxjnl-2016-208406. Epub 2016 Nov 16.

Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial.反复雾化非病毒 CFTR 基因治疗囊性纤维化患者：一项随机、双盲、安慰剂对照、2b 期试验。

Lancet Respir Med. 2015 Sep;3(9):684-691. doi: 10.1016/S2213-2600(15)00245-3. Epub 2015 Jul 3.

Cystic Fibrosis Gene Therapy in the UK and Elsewhere.英国及其他地区的囊性纤维化基因治疗

Hum Gene Ther. 2015 May;26(5):266-75. doi: 10.1089/hum.2015.027.

Assessment of F/HN-pseudotyped lentivirus as a clinically relevant vector for lung gene therapy.评估 F/HN-假型慢病毒作为一种具有临床相关性的肺部基因治疗载体。

Am J Respir Crit Care Med. 2012 Nov 1;186(9):846-56. doi: 10.1164/rccm.201206-1056OC. Epub 2012 Sep 6.

Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation.VX-770 治疗囊性纤维化患者与 G551D-CFTR 突变。

N Engl J Med. 2010 Nov 18;363(21):1991-2003. doi: 10.1056/NEJMoa0909825.

LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1.两名接受X连锁重症联合免疫缺陷（SCID-X1）基因治疗的患者中与LMO2相关的克隆性T细胞增殖。

Science. 2003 Oct 17;302(5644):415-9. doi: 10.1126/science.1088547.

Safety of a single aerosol administration of escalating doses of the cationic lipid GL-67/DOPE/DMPE-PEG5000 formulation to the lungs of normal volunteers.对正常志愿者肺部单次雾化给予递增剂量的阳离子脂质GL-67/DOPE/DMPE-PEG5000制剂的安全性。

Gene Ther. 1997 Sep;4(9):937-42. doi: 10.1038/sj.gt.3300481.

A phase I study of an adeno-associated virus-CFTR gene vector in adult CF patients with mild lung disease.一项针对患有轻度肺部疾病的成年囊性纤维化（CF）患者的腺相关病毒 - 囊性纤维化跨膜传导调节因子（CFTR）基因载体的I期研究。

Hum Gene Ther. 1996 Jun 10;7(9):1145-59. doi: 10.1089/hum.1996.7.9-1145.

文献检索

告别复杂PubMed语法，用中文像聊天一样搜索，搜遍4000万医学文献。AI智能推荐，让科研检索更轻松。

立即免费搜索

文件翻译

保留排版，准确专业，支持PDF/Word/PPT等文件格式，支持 12+语言互译。

免费翻译文档

深度研究

AI帮你快速写综述，25分钟生成高质量综述，智能提取关键信息，辅助科研写作。

立即免费体验