title: Multifunctional Oligoaminoamides for the Receptor-Specific Delivery of Therapeutic RNA.
abstract: Drugs with novel and versatile modes of action, such as therapeutic nucleic acids or proteins, open new possibilities for the precise therapy of different diseases. The most crucial limitation during the development of a therapeutic drug remains the safe and efficient intracellular delivery.To overcome the hurdles and to realize the successful delivery of such new biopharmaceuticals, our laboratory has recently developed a sequence-defined, cationic oligomer platform based on solid-phase synthesis. These multifunctional oligomers have displayed efficient delivery of therapeutic RNA in vitro and in vivo. In this chapter, we provide a brief background on the special features and applications of these carrier systems as well as detailed protocols for the oligomer and polyplex synthesis and their evaluation.
authors.affiliation: Pharmaceutical Biotechnology, Center for System-based Drug Research, Ludwig-Maximilians-University Munich, Munich, Germany.
authors.forename: Judith
authors.initials: J
authors.lastname: Weber
authors.forename: Ulrich
authors.initials: U
authors.lastname: Lächelt
authors.forename: Ernst
authors.initials: E
authors.lastname: Wagner
mesh_terms: D000577:Amides / Q000737:chemistry* / Q000378:metabolism; D000596:Amino Acids / Q000737:chemistry* / Q000378:metabolism; D000818:Animals; D016503:Drug Delivery Systems; D058973:Folate Receptors, GPI-Anchored / Q000378:metabolism; D018014:Gene Transfer Techniques; D006801:Humans; D009369:Neoplasms / Q000235:genetics / Q000378:metabolism; D010455:Peptides / Q000737:chemistry / Q000378:metabolism; D011070:Poly I-C / Q000737:chemistry / Q000378:metabolism; D034622:RNA Interference; D034741:RNA, Small Interfering / Q000008:administration & dosage* / Q000737:chemistry / Q000235:genetics / Q000378:metabolism; D000067492:RNAi Therapeutics / Q000379:methods; D060327:Solid-Phase Synthesis Techniques / Q000379:methods
chemical_list: D000577:Amides; D000596:Amino Acids; D058973:Folate Receptors, GPI-Anchored; D010455:Peptides; D034741:RNA, Small Interfering; D011070:Poly I-C