Highly efficient gene silencing and bioimaging based on fluorescent carbon dots in vitro and in vivo  

作　　者：Seongchan Kim Yuri Choi Ginam Park Cheolhee Won Young-Joon Park Younghoon Lee Byeong-Su Kim DaI-Hee Min 

机构地区：[1]Center for RNA Research, Institute for Basic Sciences (IBS), Department of Chemistry, Seoul National University, Seou108826, Republic of Korea [2]Department of Energy Engineering and Department of Chemistry, Ulsan National Institute of Science and Technology (UNIST), Ulsan 44919, Republic of Korea [3]Institute of Nanobio Convergence Technology, Lemonex Inc., Seou108826, Republic of Korea [4]College of Pharmacy, Ajou University, Worldcuplo 206, Yeongtong-gu, Suwon 16499, Republic of Korea [5]Department of Chemistry, Korea Advanced Institute of Science and Technology (KAIST), Daejeon 34141, Republic of Korea

出　　处：《Nano Research》2017年第2期503-519,共17页纳米研究（英文版）

摘　　要：Small interfering RNA （siRNA） is an attractive therapeutic candidate for sequence- specific gene silencing to treat incurable diseases using small molecule drugs. However, its efficient intracellular delivery has remained a challenge. Here, we have developed a highly biocompatible fluorescent carbon dot （CD）, and demonstrate a functional siRNA delivery system that induces efficient gene knockdown in vitro and in vivo. We found that CD nanoparticles （NPs） enhance the cellular uptake of siRNA, via endocytosis in tumor cells, with low cytotoxicity and unexpected immune responses. Real-time study of fluorescence imaging in live cells shows that CD NPs favorably localize in cytoplasm and successfully release siRNA within 12 h. Moreover, we demonstrate that CD NP-mediated siRNA delivery significantly silences green fluorescence protein （GFP） expression and inhibits tumor growth in a breast cancer cell xenograft mouse model of tumor-specific therapy. We have developed a multifunctional siRNA delivery vehicle enabling simultaneous bioimaging and efficient downregulation of gene expression, that shows excellent potential for gene therapy.Small interfering RNA （siRNA） is an attractive therapeutic candidate for sequence- specific gene silencing to treat incurable diseases using small molecule drugs. However, its efficient intracellular delivery has remained a challenge. Here, we have developed a highly biocompatible fluorescent carbon dot （CD）, and demonstrate a functional siRNA delivery system that induces efficient gene knockdown in vitro and in vivo. We found that CD nanoparticles （NPs） enhance the cellular uptake of siRNA, via endocytosis in tumor cells, with low cytotoxicity and unexpected immune responses. Real-time study of fluorescence imaging in live cells shows that CD NPs favorably localize in cytoplasm and successfully release siRNA within 12 h. Moreover, we demonstrate that CD NP-mediated siRNA delivery significantly silences green fluorescence protein （GFP） expression and inhibits tumor growth in a breast cancer cell xenograft mouse model of tumor-specific therapy. We have developed a multifunctional siRNA delivery vehicle enabling simultaneous bioimaging and efficient downregulation of gene expression, that shows excellent potential for gene therapy.

关 键 词：BIOIMAGING carbon dot gene delivery RNA interference targeted cancer therapy 

分 类 号：O[理学]