Journal article
RNAi medicine for the brain: progresses and challenges
Human molecular genetics, Vol.20(R1), pp.R21-R27
04/15/2011
DOI: 10.1093/hmg/ddr137
PMCID: PMC3095054
PMID: 21459775
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.
Details
- Title: Subtitle
- RNAi medicine for the brain: progresses and challenges
- Creators
- Ryan L Boudreau - ,Edgardo Rodríguez-Lebrón - ,Beverly L Davidson - University of Iowa
- Resource Type
- Journal article
- Publication Details
- Human molecular genetics, Vol.20(R1), pp.R21-R27
- Publisher
- Oxford University Press
- DOI
- 10.1093/hmg/ddr137
- PMID
- 21459775
- PMCID
- PMC3095054
- ISSN
- 0964-6906
- eISSN
- 1460-2083
- Language
- English
- Date published
- 04/15/2011
- Academic Unit
- Iowa Neuroscience Institute; Cardiovascular Medicine; Fraternal Order of Eagles Diabetes Research Center; Internal Medicine
- Record Identifier
- 9984065384602771
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