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用于人类基因治疗的细小病毒载体。

Parvovirus-based vectors for human gene therapy.

作者信息

Srivastava A

机构信息

Department of Medicine, Indiana University of School of Medicine, Indianapolis 46202-5120, USA.

出版信息

Blood Cells. 1994;20(2-3):531-6; discussion 536-8.

PMID:7538355
Abstract

It is becoming increasingly clear that the parvovirus-based vectors may prove to be a useful alternative to the more commonly used retroviral vectors in human gene therapy. Specifically, the adeno-associated virus 2 (AAV), a human parvovirus, has gained particular attention in view of its nonpathogenic nature as well as its remarkable site-specificity of integration into the human chromosome. Using the recombinant AAV vector system, it is feasible to obtain high-efficiency transduction of slow- or non-cycling primary hematopoietic stem and progenitor cells, without the need for prestimulation with cytokines, which could potentially lead to differentiation of these cells before transplantation.

摘要

越来越明显的是,在人类基因治疗中,基于细小病毒的载体可能被证明是比更常用的逆转录病毒载体更有用的替代品。具体而言,腺相关病毒2(AAV),一种人类细小病毒,鉴于其非致病性质以及其整合到人类染色体中的显著位点特异性而受到特别关注。使用重组AAV载体系统,可以高效转导缓慢或非循环的原代造血干细胞和祖细胞,而无需用细胞因子进行预刺激,因为细胞因子可能会在移植前导致这些细胞分化。

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1
Parvovirus-based vectors for human gene therapy.用于人类基因治疗的细小病毒载体。
Blood Cells. 1994;20(2-3):531-6; discussion 536-8.
2
Parvovirus vectors for human gene therapy.用于人类基因治疗的细小病毒载体。
Curr Opin Mol Ther. 2001 Oct;3(5):491-6.
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Targeted integration of transfected and infected adeno-associated virus vectors containing the neomycin resistance gene.靶向整合含有新霉素抗性基因的转染和感染腺相关病毒载体。
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Adeno-associated virus 2-mediated transduction and erythroid lineage-restricted expression from parvovirus B19p6 promoter in primary human hematopoietic progenitor cells.腺相关病毒2介导的转导以及细小病毒B19 p6启动子在原代人造血祖细胞中的红系谱系限制性表达。
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Chromosomal latency and expression at map unit 96 of a wild-type plus adeno-associated virus (AAV)/Neo vector and identification of p81, a new AAV transcriptional promoter.野生型腺相关病毒(AAV)/Neo载体在96图距单位处的染色体潜伏与表达以及新型AAV转录启动子p81的鉴定。
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Generation of efficient human blood progenitor-targeted recombinant adeno-associated viral vectors (AAV) by applying an AAV random peptide library on primary human hematopoietic progenitor cells.通过将腺相关病毒(AAV)随机肽文库应用于原代人造血祖细胞来生成高效的靶向人血液祖细胞的重组腺相关病毒载体(AAV)。
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Adeno-associated virus 2-mediated high efficiency gene transfer into immature and mature subsets of hematopoietic progenitor cells in human umbilical cord blood.腺相关病毒2介导的高效基因转移至人脐带血中造血祖细胞的未成熟和成熟亚群。
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Blood Cells. 1994;20(2-3):525-30.

引用本文的文献

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Influence of AAV vector tropism on long-term expression and Fc-γ receptor binding of an antibody targeting SARS-CoV-2.AAV 载体嗜性对靶向 SARS-CoV-2 的抗体的长期表达和 Fcγ 受体结合的影响。
Commun Biol. 2024 Jul 16;7(1):865. doi: 10.1038/s42003-024-06529-3.
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Characterization of wild-type adeno-associated virus type 2-like particles generated during recombinant viral vector production and strategies for their elimination.重组病毒载体生产过程中产生的野生型2型腺相关病毒样颗粒的特性及其清除策略。
J Virol. 1998 Jul;72(7):5472-80. doi: 10.1128/JVI.72.7.5472-5480.1998.
3
Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus.
在无辅助腺病毒情况下生产高滴度重组腺相关病毒载体。
J Virol. 1998 Mar;72(3):2224-32. doi: 10.1128/JVI.72.3.2224-2232.1998.
4
Adeno-associated virus type 2-mediated transfer of ecotropic retrovirus receptor cDNA allows ecotropic retroviral transduction of established and primary human cells.2型腺相关病毒介导的嗜亲性逆转录病毒受体cDNA转移可实现已建立的和原代人类细胞的嗜亲性逆转录病毒转导。
J Virol. 1997 Jul;71(7):5663-7. doi: 10.1128/JVI.71.7.5663-5667.1997.
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Parvovirus B19 promoter at map unit 6 confers autonomous replication competence and erythroid specificity to adeno-associated virus 2 in primary human hematopoietic progenitor cells.位于图谱单位6的细小病毒B19启动子赋予腺相关病毒2在原代人造血祖细胞中的自主复制能力和红系特异性。
Proc Natl Acad Sci U S A. 1995 Dec 19;92(26):12416-20. doi: 10.1073/pnas.92.26.12416.