Introduction to the special issue: traveling the intracellular highway to gene therapy.

Introduction to the special issue: traveling the intracellular highway to gene therapy.
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特刊简介:走细胞内基因治疗高速公路。

DOI:
10.1038/sj.gt.3302551
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发表时间:
2005
期刊:
影响因子:
5.1
通讯作者:
Medina-Kauwe,LK
Medina-Kauwe,LK
中科院分区:
医学3区
文献类型:
--
作者:
Medina-Kauwe,LK

文献摘要

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这个关于载体运输的特刊开始了一个简短的系列,将回顾我们目前对细胞进入,细胞内运动和基因治疗载体的核递送的知识。病毒载体的贩运,如腺病毒、腺相关病毒、慢病毒和单纯疱疹病毒,将进行审查。非病毒载体运输,包括DNA有效载荷的核递送和载体运输的细胞内屏障的定量建模,也将被覆盖。毒素使用的细胞进入途径也将被介绍,因为这些途径可以被认为是基因传递的可能替代途径,并且已经证明对各种治疗性毒素的传递是有利的。研究细胞内载体运输是改进载体设计的重要组成部分。当实验成功时,我们可能不关心载体运输的机制,但当基因表达不反映输入载体的剂量或宿主有意外反应时,理解载体-细胞相互作用成为一个重要的焦点。
This special issue on Vector Trafficking kicks off a short series that will review our current knowledge of cell entry, intracellular motility, and nuclear delivery of gene therapy vectors. Trafficking of viral vectors, such as adenovirus, adeno-associated virus, lentivirus, and herpes simplex virus, will be reviewed. Nonviral vector trafficking, including nuclear delivery of the DNA payload and quantitative modeling of intracellular barriers to vector trafficking, will also be covered. The cell entry pathways that toxins use will also be featured, as these pathways can be considered as possible alternate routes for gene delivery, and have already proven advantageous for the delivery of various therapeutic toxins.Studying intracellular vector trafficking can be an essential component of improving vector design. While we might not be concerned with the mechanisms of vector trafficking when experiments are successful, understanding vector–cell interactions becomes an important focus when gene expression does not reflect the dosage of input vector, or if the host has an unexpected response.