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非灵长类慢病毒载体。

Non-primate lentiviral vectors.

作者信息

Poeschla Eric M

机构信息

Molecular Medicine Program, Departments of Immunology and Medicine, Mayo Medical School, 200 First Street SW, Rochester, MN 55905, USA.

出版信息

Curr Opin Mol Ther. 2003 Oct;5(5):529-40.

Abstract

Feline and equine lentivirus-derived vector systems are yielding impressive preclinical results in tissue culture, animal and ex vivo human organ models. Improved basic scientific understanding of lentiviral life cycles is facilitating development of these vectors, and initial systems based on other lentiviruses have been constructed. In addition to further engineering of the individual platforms for safety and efficacy, important goals for the field are to compare different lentiviral vector systems rigorously in specific human targets, and to derive clinical grade packaging/producer cell lines. Further progress in fundamental virology will be critical. Species-specific lentiviral restriction has recently emerged as a dynamic subject relevant to lentiviral pathogenesis research and lentiviral vector-based gene therapy. This review summarizes recent results in this growing field and discusses elements of an agenda for furthering application of these vectors to human gene therapy.

摘要

猫科动物和马科动物慢病毒衍生的载体系统在组织培养、动物和离体人体器官模型中取得了令人瞩目的临床前研究成果。对慢病毒生命周期基础科学认识的提高促进了这些载体的开发,并且已经构建了基于其他慢病毒的初始系统。除了针对安全性和有效性对各个平台进行进一步工程改造外,该领域的重要目标是在特定人类靶点中严格比较不同的慢病毒载体系统,并获得临床级包装/生产细胞系。基础病毒学的进一步进展将至关重要。物种特异性慢病毒限制最近已成为与慢病毒发病机制研究和基于慢病毒载体的基因治疗相关的一个动态课题。本综述总结了这个不断发展的领域的最新成果,并讨论了推动这些载体在人类基因治疗中应用的议程要点。

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