• 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

Getting closer to hemophilia gene therapy for all?

作者信息

Reiss Ulrike M

机构信息

Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN.

出版信息

Blood Adv. 2025 Jul 22;9(14):3629-3630. doi: 10.1182/bloodadvances.2025016642.

DOI:10.1182/bloodadvances.2025016642
PMID:40658442
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12302136/
Abstract
摘要

相似文献

1
Getting closer to hemophilia gene therapy for all?所有人都能更接近血友病基因疗法了吗？
Blood Adv. 2025 Jul 22;9(14):3629-3630. doi: 10.1182/bloodadvances.2025016642.
2
Non-clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B.用于预防先天性 A 型或 B 型血友病患者出血的非凝血因子治疗方法。
Cochrane Database Syst Rev. 2024 Feb 27;2(2):CD014544. doi: 10.1002/14651858.CD014544.pub2.
3
Hemophilia Burden of Disease: A Systematic Review of the Cost-Utility Literature for Hemophilia.血友病疾病负担：血友病成本效益文献的系统评价。
J Manag Care Spec Pharm. 2018 Jul;24(7):632-642. doi: 10.18553/jmcp.2018.24.7.632.
4
Interventions for treating acute bleeding episodes in people with acquired hemophilia A.治疗获得性血友病A患者急性出血发作的干预措施。
Cochrane Database Syst Rev. 2014 Aug 28;2014(8):CD010761. doi: 10.1002/14651858.CD010761.pub2.
5
Gene therapy for hemophilia - From basic science to first approvals of "one-and-done" therapies.血友病的基因治疗——从基础科学到“一次给药，终身治愈”疗法的首次获批
Mol Ther. 2025 May 7;33(5):2015-2034. doi: 10.1016/j.ymthe.2025.03.043. Epub 2025 Mar 27.
6
Bypassing agent prophylaxis in people with hemophilia A or B with inhibitors.对患有抑制物的甲型或乙型血友病患者不进行旁路制剂预防。
Cochrane Database Syst Rev. 2017 Sep 25;9(9):CD011441. doi: 10.1002/14651858.CD011441.pub2.
7
Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B.用依特那考基因德扎帕罗韦克治疗B型血友病的基因疗法。
N Engl J Med. 2023 Feb 23;388(8):706-718. doi: 10.1056/NEJMoa2211644.
8
Human umbilical cord mesenchymal stem cell-based gene therapy for hemophilia B using scAAV-DJ/8-LP1-hFIXco transduction.基于人脐带间充质干细胞的 scAAV-DJ/8-LP1-hFIXco 转导基因治疗乙型血友病。
Stem Cell Res Ther. 2024 Jul 18;15(1):210. doi: 10.1186/s13287-024-03824-y.
9
Clotting factor concentrates given to prevent bleeding and bleeding-related complications in people with hemophilia A or B.凝血因子浓缩物用于预防甲型或乙型血友病患者的出血及与出血相关的并发症。
Cochrane Database Syst Rev. 2011 Sep 7(9):CD003429. doi: 10.1002/14651858.CD003429.pub4.
10
[Gene therapy marks the beginning of a potential "clinical cure" for hemophilia B patients].基因疗法标志着血友病B患者潜在“临床治愈”的开端。
Zhonghua Xue Ye Xue Za Zhi. 2025 May 14;46(5):385-388. doi: 10.3760/cma.j.cn121090-20250304-00111.

本文引用的文献

1
Completion of phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 years.5年以上血友病B患者etranacogene dezaparvovec基因疗法2b期试验完成。
Blood Adv. 2025 Jul 22;9(14):3543-3552. doi: 10.1182/bloodadvances.2024015291.
2
How to democratize cell and gene therapy: A global approach.如何使细胞和基因疗法民主化：一种全球方法。
Mol Ther. 2025 May 7;33(5):2082-2090. doi: 10.1016/j.ymthe.2025.03.061. Epub 2025 Apr 2.
3
Economic Burden of Haemophilia from a Societal Perspective: A Scoping Review.从社会视角看血友病的经济负担：一项范围综述
Pharmacoecon Open. 2025 Mar;9(2):179-205. doi: 10.1007/s41669-024-00540-4. Epub 2024 Nov 15.
4
Managing the challenges of paying for gene therapy: strategies for market action and policy reform in the United States.管理基因治疗支付的挑战：美国市场行动和政策改革策略。
J Comp Eff Res. 2024 Dec;13(12):e240118. doi: 10.57264/cer-2024-0118. Epub 2024 Nov 14.
5
Global seroprevalence of neutralizing antibodies against adeno-associated virus serotypes used for human gene therapies.用于人类基因治疗的腺相关病毒血清型中和抗体的全球血清流行率。
Mol Ther Methods Clin Dev. 2024 May 29;32(3):101273. doi: 10.1016/j.omtm.2024.101273. eCollection 2024 Sep 12.
6
Etranacogene dezaparvovec gene therapy for haemophilia B (HOPE-B): 24-month post-hoc efficacy and safety data from a single-arm, multicentre, phase 3 trial.用于治疗B型血友病的依特那柯基因脱衣壳病毒基因疗法（HOPE-B）：一项单臂、多中心、3期试验的24个月事后疗效和安全性数据
Lancet Haematol. 2024 Apr;11(4):e265-e275. doi: 10.1016/S2352-3026(24)00006-1. Epub 2024 Mar 1.
7
The experiences of people with haemophilia and their families of gene therapy in a clinical trial setting: regaining control, the Exigency study.临床试验环境下接受基因治疗的血友病患者及其家庭的体验：重获控制，Exigency 研究。
Orphanet J Rare Dis. 2022 Apr 4;17(1):155. doi: 10.1186/s13023-022-02256-2.
8
Global Seroprevalence of Pre-existing Immunity Against AAV5 and Other AAV Serotypes in People with Hemophilia A.全球甲型血友病患者中预先存在的抗 AAV5 及其他 AAV 血清型免疫的血清流行率。
Hum Gene Ther. 2022 Apr;33(7-8):432-441. doi: 10.1089/hum.2021.287. Epub 2022 Mar 16.
9
Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.腺相关病毒载体介导的乙型血友病基因转移。
N Engl J Med. 2011 Dec 22;365(25):2357-65. doi: 10.1056/NEJMoa1108046. Epub 2011 Dec 10.