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Hum Mol Genet. 2011 Apr 15;20(R1):R21-7. doi: 10.1093/hmg/ddr137. Epub 2011 Mar 31.

RNAi medicine for the brain: progresses and challenges.

Author information

1
Department of Internal Medicine, University of Iowa, Iowa City, IA 52242, USA. ryan-boudreau@uiowa.edu

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.

PMID:
21459775
PMCID:
PMC3095054
DOI:
10.1093/hmg/ddr137
[Indexed for MEDLINE]
Free PMC Article

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