Overcoming nonviral gene delivery barriers: perspective and future.

Overcoming nonviral gene delivery barriers: perspective and future.
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DOI:
10.1021/mp400467x
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发表时间:
2013-11-04
影响因子:
4.9
通讯作者:
Pfeifer BA
Pfeifer BA
中科院分区:
医学2区
文献类型:
--
作者:
Jones CH;Chen CK;Ravikrishnan A;Rane S;Pfeifer BA

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基因传递研究的一个关键最终目标是开发与临床相关的载体,用于对抗艾滋病等难以捉摸的疾病。尽管有很好的工程策略,但非病毒载体的效率和最终的基因调节效果受到许多体外和体内障碍的阻碍,这些障碍导致了亚病毒的表现。从这个角度来看,我们专注于与两种最常见的非病毒载体,阳离子基脂质和聚合物相关的基因传递屏障。我们提出了现有的递送障碍,并总结了目前克服这些障碍的特定媒介策略。
A key end goal of gene delivery research is to develop clinically-relevant vectors that can be used to combat elusive diseases such as AIDS. Despite promising engineering strategies, efficiency and ultimately gene modulation efficacy of nonviral vectors have been hindered by numerous in vitro and in vivo barriers that have resulted in sub-viral performance. In this perspective, we concentrate on the gene delivery barriers associated with the two most common classes of nonviral vectors, cationic-based lipids and polymers. We present the existing delivery barriers and summarize current vector-specific strategies to overcome said barriers.