title: RNA interference: mechanisms, technical challenges, and therapeutic opportunities.
abstract: The ability to inhibit gene expression via RNA interference (RNAi) has a broad therapeutic potential for various human diseases such as infections and cancers. Recent advances in mechanistic understanding of RNAi have improved the design of functional small interfering (si) RNAs with superior potency and specificity. With respect to delivery, new developments in delivery strategies have facilitated preclinical and clinical siRNA applications. This review provides valuable insights to guide the design and delivery of therapeutic siRNAs.
authors.affiliation: Department of Immunology, Institute for Cancer Research, Oslo University Hospital, Montebello, 0310, Oslo, Norway, Mouldy.Sioud@rr-research.no.
authors.forename: Mouldy
authors.initials: M
authors.lastname: Sioud
mesh_terms: D060565:Argonaute Proteins / Q000235:genetics / Q000378:metabolism; D001692:Biological Transport; D002268:Carboxypeptidases / Q000235:genetics / Q000378:metabolism; D002986:Clinical Trials as Topic; D053487:DEAD-box RNA Helicases / Q000235:genetics / Q000378:metabolism; D004337:Drug Carriers / Q000737:chemistry / Q000378:metabolism*; D055354:Exosomes / Q000378:metabolism; D006801:Humans; D008113:Liver Neoplasms / Q000235:genetics / Q000473:pathology / Q000628:therapy*; D034622:RNA Interference; D012333:RNA, Messenger / Q000037:antagonists & inhibitors / Q000235:genetics / Q000378:metabolism; D034741:RNA, Small Interfering / Q000235:genetics* / Q000378:metabolism; D043244:Ribonuclease III / Q000235:genetics / Q000378:metabolism
chemical_list: C466422:AGO2 protein, human; D060565:Argonaute Proteins; D004337:Drug Carriers; D012333:RNA, Messenger; D034741:RNA, Small Interfering; C408087:DICER1 protein, human; C517918:DROSHA protein, human; D043244:Ribonuclease III; D002268:Carboxypeptidases; C436327:SCPEP1 protein, human; D053487:DEAD-box RNA Helicases