Thai Bao Dieu Hien Kim, Kyoung-Ran Hong, Kyung Tae Voitsitskyi, Taras Lee, Jun-Seok Mao, Chengde Ahn, Dae-Ro 2024-01-19T16:01:45Z 2024-01-19T16:01:45Z 2022-01-10 2020-12 2374-7943 https://pubs.kist.re.kr/handle/201004/117728 A proper intracellular delivery method with target tissue specificity is critical to utilize the full potential of therapeutic molecules including siRNAs while minimizing their side effects. Herein, we prepare four small-sized DNA tetrahedrons (sTds) by self-assembly of different sugar backbone-modified oligonucleotides and screened them to develop a platform for kidney-targeted cytosolic delivery of siRNA. An in vivo biodistribution study revealed the kidney-specific accumulation of mirror DNA tetrahedron (L-sTd). Low opsonization of L-sTd in serum appeared to avoid liver clearance and keep its size small enough to be filtered through the glomerular basement membrane (GBM). After GBM filtration, L-sTd could be delivered into tubular cells by endocytosis. The kidney preference and the tubular cell uptake property of the mirror DNA nanostructure could be successfully harnessed for kidney-targeted intracellular delivery of p53 siRNA to treat acute kidney injury (AKI) in mice. Therefore, L-sTd could be a promising platform for kidney-targeted cytosolic delivery of siRNA to treat renal diseases. English AMER CHEMICAL SOC Kidney-Targeted Cytosolic Delivery of siRNA Using a Small-Sized Mirror DNA Tetrahedron for Enhanced Potency Article 10.1021/acscentsci.0c00763 1 ACS CENTRAL SCIENCE, v.6, no.12, pp.2250 - 2258 ACS CENTRAL SCIENCE 6 12 2250 2258 N scie scopus 000603399200016 2-s2.0-85096556737 Chemistry, Multidisciplinary Chemistry Article NANOSTRUCTURES NANOPARTICLES MEGALIN siRNA L-DNA DNA nanostructure kidney delivery