Jones Charles H, Chen Chih-Kuang, Ravikrishnan Anitha, Rane Snehal, Pfeifer Blaine A
Department of Chemical and Biological Engineering, University at Buffalo, State University of New York , Buffalo, New York, 14260-4200, United States.
Mol Pharm. 2013 Nov 4;10(11):4082-98. doi: 10.1021/mp400467x. Epub 2013 Oct 16.
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 subviral 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.
基因递送研究的一个关键最终目标是开发出可用于对抗诸如艾滋病等难治性疾病的具有临床相关性的载体。尽管有前景良好的工程策略，但非病毒载体的效率以及最终的基因调控功效一直受到众多体外和体内障碍的阻碍，这些障碍导致了低于病毒水平的性能。从这个角度来看，我们专注于与两类最常见的非病毒载体，即阳离子脂质和聚合物相关的基因递送障碍。我们介绍了现有的递送障碍，并总结了当前针对特定载体克服这些障碍的策略。
