Adeno-associated virus vectors: activity and applications in the CNS
Adeno-associated virus vectors: activity and applications in the CNS
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DOI：
10.1016/s0165-0270(00)00183-7
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发表时间：
2000-06-01
影响因子：
3
通讯作者：
Klein, RL
中科院分区：
文献类型：
--
作者：
Peel, AL;Klein, RL
转基因策略对功能研究很有用，它们也可能带来新的治疗方法。随着时间的推移，控制转基因在特定细胞群体中的表达对功能和基因治疗实验都越来越重要。腺相关病毒(AAV)载体可以为这些目的提供足够的基因表达时空控制。本文综述了利用AAV进行体内基因转移的方法。该系统的优势特征包括神经元基因表达：(1)高效；(2)长寿；(3)无毒。因此，AAV介导的基因转移是一种很好的功能基因组研究方法。20世纪90年代中后期，研究人员利用启动子和转基因的不同组合表征了载体在大脑中的活动，不断发现AAV在基础神经科学和临床神经科学中的新用途。(C)2000 Elsevier Science B.V.保留所有权利。
Transgenic strategies are useful for functional studies and they may also lead to novel therapies. Controlling transgene expression in defined cell populations over time is increasingly important for both functional and gene therapy experiments. The adeno-associated virus (AAV) vector may provide sufficient spatio-temporal control of gene expression for these purposes. This paper reviews in vivo somatic gene transfer methodology using AAV. Advantageous features of this system include neuronal gene expression that is: (1) efficient; (2) long-lived; and (3) non-toxic. Thus, AAV-mediated gene transfer is a good method for functional genomic research. From characterizing vector activity in the brain using different combinations of promoters and transgenes in the mid to late 1990s, researchers continue to discover novel uses of AAV for both basic and clinical neuroscience. (C) 2000 Elsevier Science B.V. All rights reserved.