Alhakamy Nabil A, Nigatu Adane S, Berkland Cory J, Ramsey Joshua D
Department of Pharmaceutical Chemistry, University of Kansas, Lawrence, KS, USA.
Ther Deliv. 2013 Jun;4(6):741-57. doi: 10.4155/tde.13.44.
The use of various cell-penetrating peptides (CPPs) to deliver genetic material for gene therapy applications has been a topic of interest for more than 20 years. The delivery of genetic material by using CPPs can be divided into two categories: covalently bound and electrostatically bound. Complexity of the synthesis procedure can be a significant barrier to translation when using a strategy requiring covalent binding of CPPs. In contrast, electrostatically complexing CPPs with genetic material or with a viral vector is relatively simple and has been demonstrated to improve gene delivery in both in vitro and in vivo studies. This review highlights gene therapy applications of complexes formed noncovalently between CPPs and genetic material or viruses.
二十多年来，使用各种细胞穿透肽（CPP）递送用于基因治疗的遗传物质一直是一个备受关注的话题。利用CPP递送遗传物质可分为两类：共价结合和静电结合。当使用需要CPP共价结合的策略时，合成过程的复杂性可能成为转化应用的重大障碍。相比之下，将CPP与遗传物质或病毒载体进行静电复合相对简单，并且已在体外和体内研究中均证明可改善基因递送。本综述重点介绍了CPP与遗传物质或病毒之间非共价形成的复合物在基因治疗中的应用。
