公开(公告)号：US20160208245A1

公开(公告)日：2016-07-21

申请号：US14754556

申请日：2015-06-29

Applicant: KOREA INSTITUTE OF SCIENCE AND TECHNOLOGY

Inventor： Hyung Jun AHN ,  Ick Chan KWON ,  Mihue JANG ,  Jong Hwan KIM

IPC: C12N15/113

CPC classification number: C12N15/111 ,  C12N2310/14 ,  C12N2310/3515 ,  C12N2310/531 ,  C12N2320/32

Abstract: A RNA/DNA nanoparticle for delivering siRNA where a RNA transcript including at least one hairpin structure hybridizes DNA-cholesterol conjugate and folate-DNA conjugate including a complementary sequence to the RNA transcript, and a composition including the RNA/DNA nanoparticle is provided. More specifically, because various siRNA used for different applications can be contained in the RNA/DNA nanoparticle for delivering siRNA at a high loading efficiency, and has stability to the outer attacks such as nuclease degradation. The RNA/DNA nanoparticle siRNA can be prepared by self-assembly without using polycationic agent which is harmful agent for body. The folate targeting to various cancer cells can accumulate the nanoparticle selectively on target cancer cell after intravenous injection, and make excellent gene-silencing effect inside the cancer tissue, thereby being used as a good agent for treating cancers.

Abstract translation: 提供了用于递送siRNA的RNA / DNA纳米颗粒，其中包含至少一个发夹结构的RNA转录物与DNA-胆固醇缀合物和包含与RNA转录物的互补序列的叶酸-DNA缀合物杂交，并且包含RNA / DNA纳米颗粒的组合物。 更具体地，因为用于不同应用的各种siRNA可以包含在用于以高负载效率递送siRNA的RNA / DNA纳米颗粒中，并且对外部攻击如核酸酶降解具有稳定性。 RNA / DNA纳米颗粒siRNA可以通过自组装制备，而不使用作为身体有害物质的聚阳离子。 针对各种癌细胞的叶酸可以在静脉注射后在靶癌细胞上选择性地积聚纳米颗粒，并且在癌组织内部具有优异的基因沉默效果，从而被用作治疗癌症的良好剂。