title: Novel siRNA delivery strategy: a new "strand" in CNS translational medicine?
abstract: RNA interference has been envisaged as a powerful tool for molecular and clinical investigation with a great potential for clinical applications. In recent years, increased understanding of cancer biology and stem cell biology has dramatically accelerated the development of technology for cell and gene therapy in these areas. This paper is a review of the most recent report of innovative use of siRNA to benefit several central nervous system diseases. Furthermore, a description is made of innovative strategies of delivery into the brain by means of viral and non-viral vectors with high potential for translation into clinical use. Problems are also highlighted that might hamper the transition from bench to bed, analyzing the lack of reliable preclinical models with predictive validity and the lack of effective delivery systems, which are able to overcome biological barriers and specifically reach the brain site of action.
authors.affiliation: Institute of Clinical Physiology, CNR, Siena, Italy.
authors.forename: Lisa
authors.initials: L
authors.lastname: Gherardini
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Bardi
authors.forename: Mariangela
authors.initials: M
authors.lastname: Gennaro
authors.forename: Tommaso
authors.initials: T
authors.lastname: Pizzorusso
mesh_terms: D001812:Blood-Brain Barrier / Q000378:metabolism; D004337:Drug Carriers / Q000737:chemistry; D005822:Genetic Vectors / Q000378:metabolism; D006801:Humans; D053758:Nanoparticles / Q000737:chemistry; D009422:Nervous System Diseases / Q000378:metabolism / Q000473:pathology / Q000628:therapy*; D034622:RNA Interference; D034741:RNA, Small Interfering / Q000008:administration & dosage*; D057170:Translational Research, Biomedical; D014780:Viruses / Q000235:genetics
chemical_list: D004337:Drug Carriers; D034741:RNA, Small Interfering