title: Nanotechnology for in vivo targeted siRNA delivery.
abstract: Small interfering RNAs (siRNAs) can specifically inhibit gene expression. As a result, they have tremendous scientific and clinical potential. However, the use of these molecules in patients and animal models has been limited by challenges with delivery. Intracellular RNA delivery is difficult; it requires a system that protects the siRNA from degradative nucleases in the bloodstream, minimizes clearance by the reticuloendothelial system, maximizes delivery to the target tissue, and promotes entry into, and out of, an endocytic vesicle. Despite these barriers, recent data suggest that RNA may be targeted to cells of interest in vivo. Herein we outline strategies for targeted siRNA delivery, and describe how these strategies may be improved.
authors.affiliation: The Institute for Medical Engineering and Science, Massachusetts Institute of Technology, Cambridge, MA, USA; Harvard-MIT Division of Health Sciences and Technology, Massachusetts Institute of Technology, Cambridge, MA, USA; David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, USA.
authors.forename: James E
authors.initials: JE
authors.lastname: Dahlman
authors.affiliation: Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA, USA; David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, USA.
authors.forename: Kevin J
authors.initials: KJ
authors.lastname: Kauffman
authors.affiliation: The Institute for Medical Engineering and Science, Massachusetts Institute of Technology, Cambridge, MA, USA; Harvard-MIT Division of Health Sciences and Technology, Massachusetts Institute of Technology, Cambridge, MA, USA; Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA, USA; David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, USA.
authors.forename: Robert
authors.initials: R
authors.lastname: Langer
authors.affiliation: The Institute for Medical Engineering and Science, Massachusetts Institute of Technology, Cambridge, MA, USA; Harvard-MIT Division of Health Sciences and Technology, Massachusetts Institute of Technology, Cambridge, MA, USA; Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA, USA; David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA, USA.
authors.forename: Daniel G
authors.initials: DG
authors.lastname: Anderson
mesh_terms: D000818:Animals; D016503:Drug Delivery Systems; D042783:Endothelial Cells / Q000187:drug effects; D005786:Gene Expression Regulation; D018014:Gene Transfer Techniques; D006801:Humans; D008055:Lipids / Q000008:administration & dosage / Q000737:chemistry; D008099:Liver / Q000187:drug effects / Q000502:physiology*; D053758:Nanoparticles / Q000008:administration & dosage / Q000737:chemistry*; D036103:Nanotechnology / Q000379:methods*; D009369:Neoplasms / Q000235:genetics / Q000473:pathology; D034741:RNA, Small Interfering / Q000008:administration & dosage*
chemical_list: D008055:Lipids; D034741:RNA, Small Interfering
keywords: Drug delivery; Lipid; Lipidoid; Nanoparticle; Nanotechnology; miRNA; siRNA