Sato Yusuke, Nakamura Takashi, Yamada Yuma, Akita Hidetaka, Harashima Hideyoshi
Faculty of Pharmaceutical Sciences, Hokkaido University, Sapporo City, Hokkaido, Japan.
Adv Genet. 2014;88:139-204. doi: 10.1016/B978-0-12-800148-6.00006-7.
It is anticipated that nucleic acid medicines will be in widespread use in the future, since they have the potential to cure diseases based on molecular mechanisms at the level of gene expression. However, intelligent delivery systems are required to achieve nucleic acid therapy, since they can perform their function only when they reach the intracellular site of action. We have been developing a multifunctional envelope-type nanodevice abbreviated as MEND, which consists of functional nucleic acids as a core and lipid envelope, and can control not only biodistribution but also the intracellular trafficking of nucleic acids. In this chapter, we review the development and evolution of the MEND by providing several successful examples, including the R8-MEND, the KALA-MEND, the MITO-Porter, the YSK-MEND, and the PALM.
预计核酸药物在未来将得到广泛应用,因为它们有潜力基于基因表达水平的分子机制来治愈疾病。然而,实现核酸治疗需要智能递送系统,因为核酸只有到达细胞内作用位点才能发挥其功能。我们一直在开发一种多功能包膜型纳米装置,简称为MEND,它以功能性核酸为核心,脂质为包膜,不仅可以控制核酸的生物分布,还能控制核酸在细胞内的转运。在本章中,我们通过提供几个成功的例子,包括R8-MEND、KALA-MEND、线粒体载体(MITO-Porter)、YSK-MEND和PALM,来回顾MEND的发展和演变。