title: Use of guanidinopropyl-modified siRNAs to silence gene expression.
abstract: Silencing gene expression by harnessing the RNA interference (RNAi) pathway with short interfering RNAs (siRNAs) has useful analytical and potentially therapeutic application. To augment silencing efficacy of siRNAs, chemical modification has been employed to improve stability, target specificity, and delivery to target tissues. siRNAs incorporating guanidinopropyl (GP) moieties have demonstrated enhanced target gene silencing in cell culture and in vivo models of hepatitis B virus replication. Here we describe the synthesis of GP-modified siRNAs and use of 5' rapid amplification of cDNA ends (5' RACE) to verify an RNAi-mediated mechanism of action of these novel chemically modified siRNAs.
authors.affiliation: Institute of Organic Chemistry & Chemical Biology, Goethe-University, Max-von-Laue-Str. 7, 60438, Frankfurt am Main, Germany.
authors.forename: Maximilian C R
authors.initials: MC
authors.lastname: Buff
authors.forename: Stefan
authors.initials: S
authors.lastname: Bernhardt
authors.forename: Musa D
authors.initials: MD
authors.lastname: Marimani
authors.forename: Abdullah
authors.initials: A
authors.lastname: Ely
authors.forename: Joachim W
authors.initials: JW
authors.lastname: Engels
authors.forename: Patrick
authors.initials: P
authors.lastname: Arbuthnot
mesh_terms: D045744:Cell Line, Tumor; D015870:Gene Expression; D020868:Gene Silencing; D006146:Guanidines / Q000737:chemistry; D022781:Hepatocytes / Q000166:cytology / Q000378:metabolism; D006801:Humans; D021141:Nucleic Acid Amplification Techniques; D009841:Oligonucleotides / Q000138:chemical synthesis / Q000235:genetics* / Q000378:metabolism; D009943:Organophosphorus Compounds / Q000737:chemistry; D034741:RNA, Small Interfering / Q000138:chemical synthesis* / Q000235:genetics / Q000378:metabolism; D060327:Solid-Phase Synthesis Techniques / Q000379:methods*; D014162:Transfection
chemical_list: D006146:Guanidines; D009841:Oligonucleotides; D009943:Organophosphorus Compounds; D034741:RNA, Small Interfering; C434331:phosphoramidite