title: In vivo delivery of RNAi with lipid-based nanoparticles.
abstract: RNA interference (RNAi) technology represents a fundamentally new category of treatments for human disease by addressing targets that are traditionally considered undruggable with existing medicines. The major challenge for RNAi-based therapy is the delivery system that meets human therapeutic needs. Therefore, engineering vectors with good delivery efficiency and safety profile is an intense area of research. Lipid-based nanoparticles for RNAi have yielded successful advances in vivo and to an extent in clinical trials. In this review, we discuss the barriers in developing lipid-based nanoparticles for in vivo RNAi and different strategies to overcome them. Rational designs that address safety concerns and ensure effective delivery will aid the translation of engineered lipid-based nanoparticles toward the clinic in the foreseeable future.
authors.affiliation: Division of Molecular Pharmaceutics, Eshelman School of Pharmacy, University of North Carolina at Chapel Hill, NC 27599-7571, USA. leafh@unc.edu
authors.forename: Leaf
authors.initials: L
authors.lastname: Huang
authors.forename: Yang
authors.initials: Y
authors.lastname: Liu
mesh_terms: D000465:Algorithms; D004337:Drug Carriers / Q000008:administration & dosage / Q000737:chemistry; D030342:Genetic Diseases, Inborn / Q000628:therapy; D015316:Genetic Therapy / Q000379:methods; D005822:Genetic Vectors / Q000008:administration & dosage; D006801:Humans; D008081:Liposomes / Q000008:administration & dosage / Q000737:chemistry; D053758:Nanoparticles / Q000008:administration & dosage / Q000737:chemistry; D009369:Neoplasms / Q000628:therapy; D010599:Pharmacokinetics; D011092:Polyethylene Glycols / Q000737:chemistry; D034622:RNA Interference; D034741:RNA, Small Interfering / Q000008:administration & dosage* / Q000737:chemistry; D014777:Virus Diseases / Q000628:therapy
chemical_list: D004337:Drug Carriers; D008081:Liposomes; D034741:RNA, Small Interfering; D011092:Polyethylene Glycols