title: RNAi medicine for the brain: progresses and challenges.
abstract: RNAi interference (RNAi) is a powerful gene silencing technology that has immense potential for treating a vast array of human ailments, for which suppressing disease-associated genes may provide clinical benefit. Here, we review the development of RNAi as a therapeutic modality for neurodegenerative diseases affecting the central nervous system (CNS). We overview promising preclinical data for the application of RNAi in the CNS and discuss key challenges (e.g. delivery and specificity) that remain as these approaches transition to the clinic.
authors: Department of Internal Medicine, University of Iowa, Iowa City, IA 52242, USA. ryan-boudreau@uiowa.edu
authors: Ryan L
authors: RL
authors: Boudreau
authors: Edgardo
authors: E
authors: Rodríguez-Lebrón
authors: Beverly L
authors: BL
authors: Davidson
mesh_terms: D001921:Brain / Q000473:pathology; D002490:Central Nervous System; D020868:Gene Silencing; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D008954:Models, Biological; D019636:Neurodegenerative Diseases / Q000235:genetics / Q000628:therapy*; D010455:Peptides / Q000235:genetics; D034622:RNA Interference
chemical_list: D010455:Peptides; C097188:polyglutamine