Journal article
RNAi therapeutics for CNS disorders
Brain research, Vol.1338, pp.112-121
2010
DOI: 10.1016/j.brainres.2010.03.038
PMID: 20307511
Abstract
RNA interference (RNAi) is a process of sequence-specific gene silencing and serves as a powerful molecular tool to manipulate gene expression
in vitro and
in vivo. RNAi technologies have been applied to study gene function and validate drug targets. Researchers are investigating RNAi-based compounds as novel therapeutics to treat a variety of human diseases that are currently lacking sufficient treatment. To date, numerous studies support that RNAi therapeutics can improve disease phenotypes in various rodent models of human disease. Here, we focus on the development of RNAi-based therapies aimed at treating neurological disorders for which reduction of mutant or toxic gene expression may provide clinical benefit. We review RNAi-based gene-silencing strategies, proof-of-concept studies testing therapeutic RNAi for CNS disorders, and highlight the most recent research aimed at transitioning RNAi-based therapeutics toward clinical trials.
Details
- Title: Subtitle
- RNAi therapeutics for CNS disorders
- Creators
- Ryan L Boudreau - Department of Internal Medicine, University of Iowa, Iowa City, IA, USABeverly L Davidson - Department of Internal Medicine, University of Iowa, Iowa City, IA, USA
- Resource Type
- Journal article
- Publication Details
- Brain research, Vol.1338, pp.112-121
- Publisher
- Elsevier B.V
- DOI
- 10.1016/j.brainres.2010.03.038
- PMID
- 20307511
- ISSN
- 0006-8993
- eISSN
- 1872-6240
- Language
- English
- Date published
- 2010
- Academic Unit
- Iowa Neuroscience Institute; Cardiovascular Medicine; Fraternal Order of Eagles Diabetes Research Center; Internal Medicine
- Record Identifier
- 9984065390702771
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