| Literature DB >> 21459775 |
Ryan L Boudreau1, Edgardo Rodríguez-Lebrón, Beverly L Davidson.
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.Entities:
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Year: 2011 PMID： 21459775 PMCID： PMC3095054 DOI： 10.1093/hmg/ddr137
Source DB: PubMed Journal: Hum Mol Genet ISSN： 0964-6906 Impact factor: 6.150