title: Sequence-defined oligoaminoamides for the delivery of siRNAs.
abstract: Since it was found that synthetic small interfering RNA (siRNA) can invoke RNA interference (RNAi) responses in mammalian cells, it has gained enormous attention as a tool for gene silencing in basic science and as a novel therapeutic modality. To develop carriers for cytosolic and systemic siRNA delivery, our laboratory has recently developed a sequence-defined polymer platform compatible with solid-phase-supported synthesis. These polymers have displayed efficient siRNA-mediated gene silencing in vitro and in vivo. In this chapter, we provide a brief background on the special features of these polymers and detailed protocols to evaluate polyplex formation, gene silencing efficiency, and cytotoxicity of siRNA-containing polyplexes.
authors.affiliation: Department of Pharmacy and Center for Nanoscience (CeNS), Pharmaceutical Biotechnology, Center for System-Based Drug Research, Ludwig Maximilians University of Munich, Building D, Butenandtstraße 5-13, Munich, 81377, Germany.
authors.forename: Dian-Jang
authors.initials: DJ
authors.lastname: Lee
authors.forename: Ernst
authors.initials: E
authors.lastname: Wagner
authors.forename: Taavi
authors.initials: T
authors.lastname: Lehto
mesh_terms: D000818:Animals; D002460:Cell Line; D002470:Cell Survival; D020868:Gene Silencing; D017930:Genes, Reporter; D006801:Humans; D008967:Molecular Biology / Q000379:methods*; D049329:Nanostructures; D011108:Polymers / Q000008:administration & dosage* / Q000138:chemical synthesis / Q000737:chemistry*; D034741:RNA, Small Interfering / Q000008:administration & dosage* / Q000737:chemistry / Q000378:metabolism; D060327:Solid-Phase Synthesis Techniques
chemical_list: D011108:Polymers; D034741:RNA, Small Interfering