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腺相关病毒载体作为基因递送剂:在中枢神经系统疾病和创伤中的应用。

Adeno-associated viral vectors as agents for gene delivery: application in disorders and trauma of the central nervous system.

作者信息

Ruitenberg Marc J, Eggers Ruben, Boer Gerard J, Verhaagen Joost

机构信息

Graduate School for Neurosciences Amsterdam, Netherlands Institute for Brain Research, Meibergdreef 33, 1105 AZ Amsterdam ZO, The Netherlands.

出版信息

Methods. 2002 Oct;28(2):182-94. doi: 10.1016/s1046-2023(02)00222-0.

DOI:10.1016/s1046-2023(02)00222-0
PMID:12413416
Abstract

The use of viral vectors as agents for gene delivery provides a direct approach to manipulate gene expression in the mammalian central nervous system (CNS). The present article describes in detail the methodology for the injection of viral vectors, in particular adeno-associated virus (AAV) vectors, into the adult rat brain and spinal cord to obtain reproducible and successful transduction of neural tissue. Surgical and injection procedures are based on the extensive experience of our laboratory to deliver viral vectors to the adult rat CNS and have been optimized over the years. First, a brief overview is presented on the use and potential of viral vectors to treat neurological disorders or trauma of the CNS. Next, methods to deliver AAV vectors to the rat brain and spinal cord are described in great detail with the intent of providing a practical guide to potential users. Finally, some data on the experimental outcomes following AAV vector-mediated gene transfer to the adult rat CNS are presented as is a brief discussion on both the advantages and limitations of AAV vectors as tools for somatic gene transfer.

摘要

使用病毒载体作为基因递送媒介为在哺乳动物中枢神经系统(CNS)中操纵基因表达提供了一种直接方法。本文详细描述了将病毒载体,特别是腺相关病毒(AAV)载体注射到成年大鼠脑和脊髓中以实现神经组织可重复且成功转导的方法。手术和注射程序基于我们实验室多年来向成年大鼠中枢神经系统递送病毒载体的丰富经验,并经过了多年优化。首先,简要概述了病毒载体在治疗中枢神经系统神经疾病或创伤方面的用途和潜力。接下来,详细描述了将AAV载体递送至大鼠脑和脊髓的方法,旨在为潜在用户提供实用指南。最后,给出了一些关于AAV载体介导的基因转移至成年大鼠中枢神经系统后的实验结果的数据,以及对AAV载体作为体细胞基因转移工具的优缺点的简要讨论。

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