Kim, Kyoung-Ran Jegal, Hyun Kim, Junghyun Ahn, Dae-Ro 2024-01-19T18:30:24Z 2024-01-19T18:30:24Z 2021-09-05 2020-01-21 2047-4830 https://pubs.kist.re.kr/handle/201004/119077 While siRNA is a potent therapeutic tool that can silence disease-causing mRNA, its in vivo potency can be compromised due to the lack of target tissue specificity. Here, we report a wireframe tetrahedral DNA nanostructure having a 20-mer duplex on each side that can be specifically distributed into the liver upon systemic administration. This liver-targeted DNA tetrahedron is employed as the carrier for liver-specific delivery of siRNA targeting ApoB1 mRNA, which is overexpressed in hypercholesterolemia. When delivered by a DNA tetrahedron, the siRNA can preferentially be accumulated in the liver and down-regulate the ApoB1 protein. As a result, the blood cholesterol level is also decreased by the siRNA. These results successfully demonstrate that the DNA tetrahedron is a promising carrier for liver-targeted delivery of therapeutic nucleic acids. English ROYAL SOC CHEMISTRY DRUG-DELIVERY NANOSTRUCTURES NANOPARTICLES DESIGN SYSTEM GENE A self-assembled DNA tetrahedron as a carrier for in vivo liver-specific delivery of siRNA Article 10.1039/c9bm01769k 1 BIOMATERIALS SCIENCE, v.8, no.2, pp.586 - 590 BIOMATERIALS SCIENCE 8 2 586 590 scie scopus 000509549500006 2-s2.0-85078371495 Materials Science, Biomaterials Materials Science Article DRUG-DELIVERY NANOSTRUCTURES NANOPARTICLES DESIGN SYSTEM GENE