• 文献检索
  • 文档翻译
  • 深度研究
  • 学术资讯
  • Suppr Zotero 插件Zotero 插件
  • 邀请有礼
  • 套餐&价格
  • 历史记录
应用&插件
Suppr Zotero 插件Zotero 插件浏览器插件Mac 客户端Windows 客户端微信小程序
定价
高级版会员购买积分包购买API积分包
服务
文献检索文档翻译深度研究API 文档MCP 服务
关于我们
关于 Suppr公司介绍联系我们用户协议隐私条款
关注我们

Suppr 超能文献

核心技术专利：CN118964589B侵权必究
粤ICP备2023148730 号-1Suppr @ 2026

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

The ambition for a one-and-done vision-saving AAV vector.

作者信息

Cronin Therese

机构信息

Nantes Université, CHU Nantes, INSERM, UMR1089, 44000 Nantes, France.

出版信息

Mol Ther Methods Clin Dev. 2025 Feb 3;33(1):101414. doi: 10.1016/j.omtm.2025.101414. eCollection 2025 Mar 13.

DOI:10.1016/j.omtm.2025.101414
PMID:39980804
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11840177/
Abstract
摘要

相似文献

1
The ambition for a one-and-done vision-saving AAV vector.对一次性实现挽救视力的腺相关病毒（AAV）载体的期望。
Mol Ther Methods Clin Dev. 2025 Feb 3;33(1):101414. doi: 10.1016/j.omtm.2025.101414. eCollection 2025 Mar 13.
2
The power of collective ambition.集体抱负的力量。
Harv Bus Rev. 2011 Dec;89(12):94-102, 145.
3
Functional and structural recovery of the retina after gene therapy in the RPE65 null mutation dog.RPE65基因无效突变犬基因治疗后视网膜的功能和结构恢复
Invest Ophthalmol Vis Sci. 2003 Apr;44(4):1663-72. doi: 10.1167/iovs.02-0595.
4
Screening of a retinal-targeting Adeno-Associated Virus (AAV) via DNA shuffling.通过DNA改组筛选视网膜靶向腺相关病毒（AAV）
Exp Eye Res. 2025 Feb;251:110245. doi: 10.1016/j.exer.2025.110245. Epub 2025 Jan 21.
5
Evaluation of AAV-DJ vector for retinal gene therapy.用于视网膜基因治疗的腺相关病毒-DJ载体评估
PeerJ. 2019 Jan 17;7:e6317. doi: 10.7717/peerj.6317. eCollection 2019.
6
Intraocular route of AAV2 vector administration defines humoral immune response and therapeutic potential.AAV2载体眼内给药途径决定体液免疫反应和治疗潜力。
Mol Vis. 2008 Sep 24;14:1760-9.
7
S/MAR Element Facilitates Episomal Long-Term Persistence of Adeno-Associated Virus Vector Genomes in Proliferating Cells.S/MAR 元件促进腺相关病毒载体基因组在增殖细胞中的游离体长期持久性。
Hum Gene Ther. 2017 Dec;28(12):1169-1179. doi: 10.1089/hum.2017.025. Epub 2017 Jun 29.
8
A Single Vector Platform for High-Level Gene Transduction of Central Neurons: Adeno-Associated Virus Vector Equipped with the Tet-Off System.用于中枢神经元高效基因转导的单一载体平台：配备Tet-Off系统的腺相关病毒载体
PLoS One. 2017 Jan 6;12(1):e0169611. doi: 10.1371/journal.pone.0169611. eCollection 2017.
9
Inverted terminal repeat sequences are important for intermolecular recombination and circularization of adeno-associated virus genomes.反向末端重复序列对于腺相关病毒基因组的分子间重组和环化很重要。
J Virol. 2005 Jan;79(1):364-79. doi: 10.1128/JVI.79.1.364-379.2005.
10
Extending AAV Packaging Cargo through Dual Co-Transduction: Efficient Protein Trans-Splicing at Low Vector Doses.通过双重共转导扩展 AAV 包装载量：低载体剂量下的高效蛋白转剪接。
Int J Mol Sci. 2023 Jun 23;24(13):10524. doi: 10.3390/ijms241310524.

本文引用的文献

1
Preclinical evaluation of NG101, a potential AAV gene therapy for wet age-related macular degeneration.NG101的临床前评估，一种用于湿性年龄相关性黄斑变性的潜在腺相关病毒基因疗法。
Mol Ther Methods Clin Dev. 2024 Nov 5;32(4):101366. doi: 10.1016/j.omtm.2024.101366. eCollection 2024 Dec 12.
2
Gene therapy for neovascular age-related macular degeneration by subretinal delivery of RGX-314: a phase 1/2a dose-escalation study.经视网膜下腔递送达 RGX-314 治疗新生血管性年龄相关性黄斑变性的基因治疗：1/2a 期剂量递增研究。
Lancet. 2024 Apr 20;403(10436):1563-1573. doi: 10.1016/S0140-6736(24)00310-6. Epub 2024 Mar 27.
3
Intravitreal aflibercept 8 mg in neovascular age-related macular degeneration (PULSAR): 48-week results from a randomised, double-masked, non-inferiority, phase 3 trial.玻璃体内注射阿柏西普 8mg 治疗新生血管性年龄相关性黄斑变性（PULSAR）：一项随机、双盲、非劣效性、3 期临床试验的 48 周结果。
Lancet. 2024 Mar 23;403(10432):1141-1152. doi: 10.1016/S0140-6736(24)00063-1. Epub 2024 Mar 7.
4
Safety and Efficacy of Adeno-Associated Viral Gene Therapy in Patients With Retinal Degeneration: A Systematic Review and Meta-Analysis.腺相关病毒基因治疗在视网膜变性患者中的安全性和疗效：系统评价和荟萃分析。
Transl Vis Sci Technol. 2023 Nov 1;12(11):24. doi: 10.1167/tvst.12.11.24.
5
Delayed anti-VEGF injections during the COVID-19 pandemic and changes in visual acuity in patients with three common retinal diseases: A systematic review and meta-analysis.COVID-19 大流行期间抗 VEGF 注射延迟与三种常见视网膜疾病患者视力变化的系统评价和荟萃分析。
Surv Ophthalmol. 2022 Nov-Dec;67(6):1593-1602. doi: 10.1016/j.survophthal.2022.08.002. Epub 2022 Aug 13.
6
The Effect of Treatment Discontinuation During the COVID-19 Pandemic on Visual Acuity in Exudative Neovascular Age-Related Macular Degeneration: 1-Year Results.COVID-19大流行期间治疗中断对渗出性年龄相关性黄斑变性患者视力的影响：1年结果
Ophthalmol Ther. 2021 Dec;10(4):935-945. doi: 10.1007/s40123-021-00381-y. Epub 2021 Aug 9.
7
Circumventing cellular immunity by miR142-mediated regulation sufficiently supports rAAV-delivered OVA expression without activating humoral immunity.通过 miR142 介导的调节来规避细胞免疫足以支持 rAAV 递送的 OVA 表达，而不会激活体液免疫。
JCI Insight. 2019 May 21;5(13):99052. doi: 10.1172/jci.insight.99052.
8
AAV8 Can Induce Innate and Adaptive Immune Response in the Primate Eye.AAV8 可在灵长类动物眼中诱导先天和适应性免疫反应。
Mol Ther. 2017 Dec 6;25(12):2648-2660. doi: 10.1016/j.ymthe.2017.08.018. Epub 2017 Aug 31.
9
Phase 2a Randomized Clinical Trial: Safety and Post Hoc Analysis of Subretinal rAAV.sFLT-1 for Wet Age-related Macular Degeneration.2a期随机临床试验：视网膜下注射重组腺相关病毒.sFLT-1治疗湿性年龄相关性黄斑变性的安全性及事后分析
EBioMedicine. 2016 Dec;14:168-175. doi: 10.1016/j.ebiom.2016.11.016. Epub 2016 Nov 10.
10
Global prevalence of age-related macular degeneration and disease burden projection for 2020 and 2040: a systematic review and meta-analysis.全球与年龄相关的黄斑变性患病率及 2020 与 2040 年疾病负担预测：系统回顾和荟萃分析。
Lancet Glob Health. 2014 Feb;2(2):e106-16. doi: 10.1016/S2214-109X(13)70145-1. Epub 2014 Jan 3.