基因转移至中枢神经系统的方法。
Methods for gene transfer to the central nervous system.
作者信息
Kantor Boris, Bailey Rachel M, Wimberly Keon, Kalburgi Sahana N, Gray Steven J
机构信息
Department of Pharmacology, Physiology, and Neuroscience, University of South Carolina, Columbia, SC, USA.
Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
出版信息
Adv Genet. 2014;87:125-97. doi: 10.1016/B978-0-12-800149-3.00003-2.
Gene transfer is an increasingly utilized approach for research and clinical applications involving the central nervous system (CNS). Vectors for gene transfer can be as simple as an unmodified plasmid, but more commonly involve complex modifications to viruses to make them suitable gene delivery vehicles. This chapter will explain how tools for CNS gene transfer have been derived from naturally occurring viruses. The current capabilities of plasmid, retroviral, adeno-associated virus, adenovirus, and herpes simplex virus vectors for CNS gene delivery will be described. These include both focal and global CNS gene transfer strategies, with short- or long-term gene expression. As is described in this chapter, an important aspect of any vector is the cis-acting regulatory elements incorporated into the vector genome that control when, where, and how the transgene is expressed.
基因转移是一种在涉及中枢神经系统（CNS）的研究和临床应用中越来越常用的方法。基因转移载体可以像未修饰的质粒一样简单，但更常见的是对病毒进行复杂修饰，使其成为合适的基因递送载体。本章将解释中枢神经系统基因转移工具是如何从天然存在的病毒衍生而来的。将描述质粒、逆转录病毒、腺相关病毒、腺病毒和单纯疱疹病毒载体用于中枢神经系统基因递送的当前能力。这些包括局部和整体中枢神经系统基因转移策略，以及短期或长期基因表达。如本章所述，任何载体的一个重要方面是整合到载体基因组中的顺式作用调控元件，这些元件控制转基因何时、何地以及如何表达。
Zotero 插件