title: Functional nanostructures for effective delivery of small interfering RNA therapeutics.
abstract: Small interfering RNA (siRNA) has proved to be a powerful tool for target-specific gene silencing via RNA interference (RNAi). Its ability to control targeted gene expression gives new hope to gene therapy as a treatment for cancers and genetic diseases. However, siRNA shows poor pharmacological properties, such as low serum stability, off-targeting, and innate immune responses, which present a significant challenge for clinical applications. In addition, siRNA cannot cross the cell membrane for RNAi activity because of its anionic property and stiff structure. Therefore, the development of a safe, stable, and efficient system for the delivery of siRNA therapeutics into the cytoplasm of targeted cells is crucial. Several nanoparticle platforms for siRNA delivery have been developed to overcome the major hurdles facing the therapeutic uses of siRNA. This review covers a broad spectrum of non-viral siRNA delivery systems developed for enhanced cellular uptake and targeted gene silencing in vitro and in vivo and discusses their characteristics and opportunities for clinical applications of therapeutic siRNA.
authors.affiliation: 1. Department of Biological Sciences, Korea Advanced Institute of Science and Technology (KAIST), 291 Daehak-ro, Yuseong-gu, Daejeon, 305-701, Republic of Korea;
authors.forename: Cheol Am
authors.initials: CA
authors.lastname: Hong
authors.affiliation: 1. Department of Biological Sciences, Korea Advanced Institute of Science and Technology (KAIST), 291 Daehak-ro, Yuseong-gu, Daejeon, 305-701, Republic of Korea; ; 2. Department of Materials Science and Engineering, Korea Advanced Institute of Science and Technology (KAIST), 291 Daehak-ro, Yuseong-gu, Daejeon, 305-701, Republic of Korea; ; 3. KAIST Institute for NanoCentury and BioCentury, Korea Advanced Institute of Science and Technology (KAIST), 291 Daehak-ro, Yuseong-gu, Daejeon, 305-701, Republic of Korea.
authors.forename: Yoon Sung
authors.initials: YS
authors.lastname: Nam
mesh_terms: D000818:Animals; D016503:Drug Delivery Systems / Q000295:instrumentation / Q000379:methods*; D015316:Genetic Therapy; D006801:Humans; D049329:Nanostructures / Q000737:chemistry* / Q000627:therapeutic use; D009369:Neoplasms / Q000235:genetics* / Q000378:metabolism / Q000628:therapy; D034622:RNA Interference; D034741:RNA, Small Interfering / Q000737:chemistry / Q000235:genetics* / Q000378:metabolism / Q000627:therapeutic use
chemical_list: D034741:RNA, Small Interfering
keywords: gene delivery; gene silencing; nanoparticles; non-viral vectors; small interfering RNA (siRNA).