Chowdhury E H
International Medical University, School of Medicine, No. 126, Jalan 19/155B, Bukit Jalil 57000, Kuala Lumpur, Malaysia.
Expert Opin Drug Deliv. 2009 Jul;6(7):697-703. doi: 10.1517/17425240903025744.
The nuclear envelope presents a major barrier to transgene delivery and expression using a non-viral vector. Virus is capable of overcoming the barrier to deliver their genetic materials efficiently into the nucleus by virtue of the specialized protein components with the unique amino acid sequences recognizing cellular nuclear transport machinery. However, considering the safety issues in the clinical gene therapy for treating critical human diseases, non-viral systems are highly promising compared with their viral counterparts. This review summarizes the progress on exploring the nuclear traffic mechanisms for the prominent viral vectors and the technological innovations for the nuclear delivery of non-viral DNA by mimicking those natural processes evolved for the viruses as well as for many cellular proteins.
核膜对使用非病毒载体进行转基因递送和表达构成了主要障碍。病毒能够凭借具有识别细胞核转运机制的独特氨基酸序列的特殊蛋白质成分,克服这一障碍,将其遗传物质高效递送至细胞核。然而,考虑到在治疗严重人类疾病的临床基因治疗中的安全问题,与病毒系统相比,非病毒系统具有很大的前景。本综述总结了在探索主要病毒载体的核运输机制方面的进展,以及通过模仿病毒和许多细胞蛋白所进化出的天然过程实现非病毒DNA核递送的技术创新。