腺相关病毒与肝细胞：亦敌亦友？ - Suppr

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超能文献

Adeno-associated virus and hepatocytes: frenemies?

作者信息

Pierce Glenn F

机构信息

World Federation of Hemophilia, Montreal, Canada.

出版信息

Blood Adv. 2024 Oct 8;8(19):5200-5202. doi: 10.1182/bloodadvances.2024013845.

DOI:10.1182/bloodadvances.2024013845

PMID:39378030

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11530391/

Abstract

摘要

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e4d1/11530391/c76d912f3f95/BLOODA_ADV-2024-013845-C-gr1.jpg

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本文引用的文献

Optimizing liver health before and after gene therapy for hemophilia A.优化血友病 A 基因治疗前后的肝脏健康。

Blood Adv. 2024 Oct 8;8(19):5203-5212. doi: 10.1182/bloodadvances.2024013059.

Deciphering conundrums of adeno-associated virus liver-directed gene therapy: focus on hemophilia.解读腺相关病毒肝脏定向基因治疗的难题：关注血友病。

J Thromb Haemost. 2024 May;22(5):1263-1289. doi: 10.1016/j.jtha.2023.12.005. Epub 2023 Dec 14.

Cellular stress and coagulation factor production: when more is not necessarily better.细胞应激与凝血因子生成：过犹不及。

J Thromb Haemost. 2023 Dec;21(12):3329-3341. doi: 10.1016/j.jtha.2023.10.005. Epub 2023 Oct 14.

Interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia A.腺相关病毒基因治疗血友病 A 后转导 mRNA 和蛋白产生的个体间变异性。

Nat Med. 2022 Apr;28(4):789-797. doi: 10.1038/s41591-022-01751-0. Epub 2022 Apr 11.

Evolutionary insights into coagulation factor IX Padua and other high-specific-activity variants.凝血因子 IX Padua 和其他高比活变体的进化见解。

Blood Adv. 2021 Mar 9;5(5):1324-1332. doi: 10.1182/bloodadvances.2019000405.

Immune Responses to Viral Gene Therapy Vectors.病毒基因治疗载体的免疫反应。

Mol Ther. 2020 Mar 4;28(3):709-722. doi: 10.1016/j.ymthe.2020.01.001. Epub 2020 Jan 10.

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.

Progression to end-stage liver disease in patients with inherited bleeding disorders and hepatitis C: an international, multicenter cohort study.遗传性出血性疾病合并丙型肝炎患者进展至终末期肝病：一项国际多中心队列研究。

Blood. 2007 May 1;109(9):3667-71. doi: 10.1182/blood-2006-08-038349. Epub 2007 Jan 9.

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response.腺相关病毒介导的因子IX对血友病患者肝脏的成功转导及宿主免疫反应带来的限制

Nat Med. 2006 Mar;12(3):342-7. doi: 10.1038/nm1358. Epub 2006 Feb 12.

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