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lentiviral gene therapy for severe hemophilia A: an alternative to recombinant adeno-associated viral-based strategies?

作者信息

Samelson-Jones Benjamin J

机构信息

The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

Department of Pediatrics, Division of Hematology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.

出版信息

Res Pract Thromb Haemost. 2025 Mar 20;9(2):102716. doi: 10.1016/j.rpth.2025.102716. eCollection 2025 Feb.

DOI:10.1016/j.rpth.2025.102716
PMID:40212772
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11982970/
Abstract
摘要

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Int J Mol Sci. 2024 Jan 19;25(2):1267. doi: 10.3390/ijms25021267.
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本文引用的文献

1
Platelet-Targeted Gene Therapy for Hemophilia A with Inhibitor History.针对有抑制剂病史的甲型血友病的血小板靶向基因疗法。
N Engl J Med. 2025 Jan 23;392(4):412-414. doi: 10.1056/NEJMc2415164.
2
Lentiviral Gene Therapy with CD34+ Hematopoietic Cells for Hemophilia A.采用CD34+造血细胞的慢病毒基因疗法治疗甲型血友病
N Engl J Med. 2025 Jan 30;392(5):450-457. doi: 10.1056/NEJMoa2410597. Epub 2024 Dec 9.
3
Pre-clinical evaluation of an enhanced-function factor VIII variant for durable hemophilia A gene therapy in male mice.增强功能因子 VIII 变异体在雄性小鼠中用于持久血友病 A 基因治疗的临床前评估。
Nat Commun. 2024 Aug 21;15(1):7193. doi: 10.1038/s41467-024-51296-8.
4
Coagulation factor VIII: biological basis of emerging hemophilia A therapies.凝血因子 VIII：新兴血友病 A 治疗方法的生物学基础。
Blood. 2024 Nov 21;144(21):2185-2197. doi: 10.1182/blood.2023023275.
5
Roctavian gene therapy for hemophilia A.罗氏血友病 A 的基因治疗药物。
Blood Adv. 2024 Oct 8;8(19):5179-5189. doi: 10.1182/bloodadvances.2023011847.
6
Platelet-targeted gene therapy induces immune tolerance in hemophilia and beyond.血小板靶向基因治疗诱导血友病及其他疾病的免疫耐受。
J Thromb Haemost. 2024 Jan;22(1):23-34. doi: 10.1016/j.jtha.2023.07.025. Epub 2023 Aug 7.
7
A review of the rationale for gene therapy for hemophilia A with inhibitors: one-shot tolerance and treatment?血友病 A 伴抑制物基因治疗的合理性回顾：一针性耐受与治疗？
J Thromb Haemost. 2023 Nov;21(11):3033-3044. doi: 10.1016/j.jtha.2023.05.011. Epub 2023 May 22.
8
Adeno-Associated Virus Gene Therapy for Hemophilia.腺相关病毒基因治疗血友病。
Annu Rev Med. 2023 Jan 27;74:231-247. doi: 10.1146/annurev-med-043021-033013. Epub 2022 Sep 14.
9
Living with a "hemophilia-free mind" - The new ambition of hemophilia care?以“无血友病心态”生活——血友病护理的新目标？
Res Pract Thromb Haemost. 2021 Aug 3;5(5):e12567. doi: 10.1002/rth2.12567. eCollection 2021 Jul.
10
Non-genotoxic conditioning facilitates hematopoietic stem cell gene therapy for hemophilia A using bioengineered factor VIII.非基因毒性预处理有助于利用生物工程因子VIII对A型血友病进行造血干细胞基因治疗。
Mol Ther Methods Clin Dev. 2021 May 5;21:710-727. doi: 10.1016/j.omtm.2021.04.016. eCollection 2021 Jun 11.