title: [SiRNA-based therapeutics].
abstract: Since the discovery of RNAi in worms in 1998 that earned the Nobel Prize in 2006, the race continues in the effort to realize RNAi-based treatment in clinics. The major challenge for the realization of RNAi-based medicine is, however, to direct intact RNAs to the right tissues in the body and then successfully escort them into cells without eliciting off-target effects and unwanted immune stimulation. Novel technologies have been developed that enable systemic and targeted siRNA delivery, inducing robust and specific therapeutic gene silencing in vivo.
authors.affiliation: Program of Cellular and Molecular Medicine, Children's Hospital, Boston, USA.
authors.forename: Motomu
authors.initials: M
authors.lastname: Shimaoka
mesh_terms: D020868:Gene Silencing; D006801:Humans; D034622:RNA Interference; D034741:RNA, Small Interfering / Q000008:administration & dosage / Q000627:therapeutic use
chemical_list: D034741:RNA, Small Interfering