Study on the innovative molecular-targeting medicine based on the nano-DDS encapsulating intelligent artificial oligonucleotides

核酸药物综合研究项目已定向于靶向致病机理的纳米药物。通过相互合作，在人工功能性寡核苷酸的化学研究、靶基因的确定、选择性靶向细胞或组织的递送系统以及纳米药物的模型动物验证等方面积累了成果。新开发的反应性寡糖肽（reactive oligonulceotides， ODN）被包裹在β - mend中，靶向β细胞，通过miRNA-375系统将其递送至β细胞系的MIN6细胞，从而释放胰岛素的天然抑制作用，使胰岛素升高。细胞凋亡抑制剂Bonkrekic酸通过在线粒体靶向递送系统MITO-Porter中包封而增强其活性。将靶向Drp1基因的siRNA包被在YSK-MEND（肝脏靶向纳米包膜）中，以验证其在模型动物中的作用，并提出了一种新的治疗方法。

Integrated research project for nucleic acids drug has been directed to nano-medicines that can target pathogenetic geneses. Achievements on chemical research for the artificial functional oligonucleotides, determination of the target genes, delivery system for selective targeting cells or tissues, and validation of the nano-medicine using model animals have been accumulates by mutual collaboration.Newly-developed reactive oligonulceotides (ODN) are encapsulated in beta-MEND to target beta-cells, which are delivered into MIN6 cells of beta cell line, resulting in increase of insulin by releasing its native inhibition by miRNA-375 system. Bonkrekic acid, an apoptosis inhibitor, enhanced its activity by encapsulation in MITO-Porter, a mitochondria- targeting delivery system. siRNA targeting Drp1 gene was encapsulated in YSK-MEND (liver-targeting nano-envelope) for validate its effect for the model animal, and a potential as a new therapeutic approach has been suggested.