title: siRNA delivery: from basics to therapeutic applications.
abstract: The chance to selectively intervene and stop the development of any gene-dependent disease in different organs and pathologies makes siRNA an ideal therapeutic agent. However, serious issues should be addressed before the real therapeutic use of siRNA. The poor pharmacokinetic properties of siRNA, its short half-life, its low in vivo stability, its fast elimination by kidney filtration and its low transfection efficiency complicate the use of siRNA as a therapeutic molecule. In this review, we will describe the latest and most advanced approaches and strategies undertaken to address these limitations and improve siRNA delivery and its gene silencing efficacy as well as the prospects for its therapeutic applications.
authors.affiliation: Center for Pharmaceutical Biotechnology and Nanomedicine, Northeastern University, 360 Huntington Ave, Boston, MA 02115, USA.
authors.forename: Tiziana
authors.initials: T
authors.lastname: Musacchio
authors.forename: Vladimir P
authors.initials: VP
authors.lastname: Torchilin
mesh_terms: D012321:DNA-Directed RNA Polymerases / Q000378:metabolism; D016503:Drug Delivery Systems / Q000379:methods; D004355:Drug Stability; D006801:Humans; D008823:Micelles; D053758:Nanoparticles / Q000008:administration & dosage; D011092:Polyethylene Glycols / Q000008:administration & dosage; D034622:RNA Interference; D012333:RNA, Messenger / Q000037:antagonists & inhibitors; D034741:RNA, Small Interfering / Q000008:administration & dosage / Q000627:therapeutic use*; D014764:Viral Proteins / Q000378:metabolism
chemical_list: D008823:Micelles; D012333:RNA, Messenger; D034741:RNA, Small Interfering; D014764:Viral Proteins; D011092:Polyethylene Glycols; C062210:bacteriophage T7 RNA polymerase; D012321:DNA-Directed RNA Polymerases