title: Cell penetrating peptide-mediated systemic siRNA delivery to the liver.
abstract: The cell-penetrating peptide (CPP) is one of the most attractive tools for efficiently delivering biomolecules to a target organelle. Here, we describe the use of octaarginine (R8)-modified lipid nanoparticles for the efficient and targeted in vivo delivery of siRNA to the liver. In this study, SR-BI (a scavenger receptor class B, member 1) was targeted by this nanoparticle. Our results demonstrate that R8-modified lipid nanoparticles can be used for the efficient and targeted delivery of liver siRNA to induce the specific knock-down of an endogenous gene with minimum liver toxicity and immune response, and that this CPP based technology holds considerable promise for further in vivo biological applications of siRNA.
authors.affiliation: Graduate School of Pharmaceutical Sciences, Hokkaido University, Sapporo, Hokkaido, Japan.
authors.forename: Yasuhiro
authors.initials: Y
authors.lastname: Hayashi
authors.forename: Jun
authors.initials: J
authors.lastname: Yamauchi
authors.forename: Ikramy A
authors.initials: IA
authors.lastname: Khalil
authors.forename: Kazuaki
authors.initials: K
authors.lastname: Kajimoto
authors.forename: Hidetaka
authors.initials: H
authors.lastname: Akita
authors.forename: Hideyoshi
authors.initials: H
authors.lastname: Harashima
mesh_terms: D000818:Animals; D018955:CD36 Antigens / Q000235:genetics; D057846:Cell-Penetrating Peptides / Q000737:chemistry*; D005260:Female; D055785:Gene Knockdown Techniques; D018014:Gene Transfer Techniques; D008055:Lipids / Q000737:chemistry; D008099:Liver / Q000378:metabolism*; D008297:Male; D051379:Mice; D008810:Mice, Inbred C57BL; D008813:Mice, Inbred ICR; D053758:Nanoparticles; D009842:Oligopeptides / Q000737:chemistry; D034741:RNA, Small Interfering / Q000008:administration & dosage* / Q000633:toxicity
chemical_list: D018955:CD36 Antigens; D057846:Cell-Penetrating Peptides; D008055:Lipids; D009842:Oligopeptides; D034741:RNA, Small Interfering; C448619:octaarginine