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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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Optimizing liver health before and after gene therapy for hemophilia A.

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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.

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.

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.

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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.

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.

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.

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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

相似文献

Adeno-associated virus and hepatocytes: frenemies?

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

Enhanced transgene expression from recombinant single-stranded D-sequence-substituted adeno-associated virus vectors in human cell lines in vitro and in murine hepatocytes in vivo.

J Virol. 2015 Jan 15;89(2):952-61. doi: 10.1128/JVI.02581-14. Epub 2014 Oct 29.

Pristimerin enhances recombinant adeno-associated virus vector-mediated transgene expression in human cell lines in vitro and murine hepatocytes in vivo.

J Integr Med. 2014 Jan;12(1):20-34. doi: 10.1016/S2095-4964(14)60003-0.

Adeno-associated virus vectors.

Curr Opin Biotechnol. 1992 Oct;3(5):533-9. doi: 10.1016/0958-1669(92)90082-t.

Hepatocyte Heparan Sulfate Is Required for Adeno-Associated Virus 2 but Dispensable for Adenovirus 5 Liver Transduction In Vivo.

J Virol. 2015 Oct 21;90(1):412-20. doi: 10.1128/JVI.01939-15. Print 2016 Jan 1.

Human Hepatocyte Transduction with Adeno-Associated Virus Vector.

Methods Mol Biol. 2022;2544:83-93. doi: 10.1007/978-1-0716-2557-6_5.

Hum Gene Ther. 2010 Mar;21(3):271-83. doi: 10.1089/hum.2009.100.

A limited number of transducible hepatocytes restricts a wide-range linear vector dose response in recombinant adeno-associated virus-mediated liver transduction.

J Virol. 2002 Nov;76(22):11343-9. doi: 10.1128/jvi.76.22.11343-11349.2002.

Strategies for improving the transduction efficiency of single-stranded adeno-associated virus vectors in vitro and in vivo.

Gene Ther. 2008 Sep;15(18):1287-93. doi: 10.1038/gt.2008.89. Epub 2008 May 22.

Adeno-associated virus as a vector for liver-directed gene therapy.

J Virol. 1998 Dec;72(12):10222-6. doi: 10.1128/JVI.72.12.10222-10226.1998.

本文引用的文献

Optimizing liver health before and after gene therapy for hemophilia A.

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.

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.

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.

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.

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

Immune Responses to Viral Gene Therapy Vectors.

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.

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.

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.

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