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.
Abstract
摘要
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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.
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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.
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