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逆转录病毒载体介导的基因治疗的最新进展:让旧载体发挥新作用

Recent advances in retrovirus vector-mediated gene therapy: teaching an old vector new tricks.

作者信息

Weber E, Anderson W F, Kasahara N

机构信息

USC Institute for Genetic Medicine, Los Angeles, CA 90033, USA.

出版信息

Curr Opin Mol Ther. 2001 Oct;3(5):439-53.

PMID:11699888
Abstract

Oncoretrovirus-based vectors have been shown to be a safe and reliable vector system that can achieve permanent integration of delivered transgenes. Successful application of these vectors for gene therapy has proven difficult due to their relatively low transduction efficiency; however, cumulative improvements in methodology have recently yielded promising clinical results. Furthermore, significant improvements in basic retrovirus vector technology now promise to revitalize the field. This review focuses on these important recent developments in the field of retrovirus-mediated gene transfer technology and its application to human diseases.

摘要

基于致癌性逆转录病毒的载体已被证明是一种安全可靠的载体系统,能够实现所传递转基因的永久整合。由于其相对较低的转导效率,这些载体在基因治疗中的成功应用已被证明具有挑战性;然而,方法学上的累积改进最近已产生了有前景的临床结果。此外,基本逆转录病毒载体技术的重大改进现在有望振兴该领域。本综述重点关注逆转录病毒介导的基因转移技术领域这些重要的最新进展及其在人类疾病中的应用。

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Recent advances in retrovirus vector-mediated gene therapy: teaching an old vector new tricks.逆转录病毒载体介导的基因治疗的最新进展:让旧载体发挥新作用
Curr Opin Mol Ther. 2001 Oct;3(5):439-53.
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Gene transfer into hematopoietic progenitor and stem cells: progress and problems.基因导入造血祖细胞和干细胞:进展与问题
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Fetal gene therapy: opportunities and risks.胎儿基因治疗:机遇与风险。
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Development of retroviral vectors for gene transfer to airway epithelia.用于将基因导入气道上皮细胞的逆转录病毒载体的研发
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Retroviral vectors for gene therapy of AIDS and cancer.用于艾滋病和癌症基因治疗的逆转录病毒载体。
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Progress towards hematopoietic stem cell gene therapy.造血干细胞基因治疗的进展。
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Retroviral vector-mediated gene expression in hematopoietic cells.逆转录病毒载体介导的造血细胞基因表达。
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Long-term gene expression in dividing and nondividing cells using SV40-derived vectors.
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Dicistronic MLV-retroviral vectors transduce neural precursors in vivo and co-express two genes in their differentiated neuronal progeny.双顺反子莫洛尼氏鼠白血病病毒逆转录病毒载体在体内转导神经前体细胞,并在其分化的神经元后代中共表达两个基因。
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Multiple modifications allow high-titer production of retroviral vectors carrying heterologous regulatory elements.多种修饰可实现携带异源调控元件的逆转录病毒载体的高滴度生产。
J Virol. 2004 Feb;78(3):1384-92. doi: 10.1128/jvi.78.3.1384-1392.2004.
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