Journal article
RNAi therapy for neurodegenerative diseases
Current topics in developmental biology, Vol.75, pp.73-92
2006
DOI: 10.1016/S0070-2153(06)75003-7
PMID: 16984810
Abstract
RNA interference (RNAi) mediates gene silencing in a sequence-specific manner and has proven to be an exceptionally valuable discovery for bench scientists. In the laboratory, RNAi technologies provide efficient means for validating drug targets and for performing reverse genetics to study gene function (Friedman and Perrimon, 2004). Patients may also benefit from RNAi as applications extend to potential human therapies. RNAi-based treatments are being investigated and may provide hope for patients suffering from cancer, viral infections, or genetic diseases for which effective therapies are currently lacking. Notably, several independent studies have demonstrated that RNAi therapy can improve disease phenotypes in various mouse models of human disease. In this chapter, we focus on the potential of RNAi in treating neurologic diseases for which reduction of mutant or toxic gene expression may provide therapeutic benefit. We discuss approaches to achieving RNAi in vivo, progress in the field, and the potential pitfalls associated with RNAi-based therapies.
Details
- Title: Subtitle
- RNAi therapy for neurodegenerative diseases
- Creators
- Ryan L Boudreau - Program in Gene Therapy, Department of Internal Medicine, University of Iowa College of Medicine, Iowa City, Iowa 52242, USABeverly L Davidson
- Resource Type
- Journal article
- Publication Details
- Current topics in developmental biology, Vol.75, pp.73-92
- Publisher
- United States
- DOI
- 10.1016/S0070-2153(06)75003-7
- PMID
- 16984810
- ISSN
- 0070-2153
- eISSN
- 1557-8933
- Language
- English
- Date published
- 2006
- Academic Unit
- Iowa Neuroscience Institute; Cardiovascular Medicine; Fraternal Order of Eagles Diabetes Research Center; Internal Medicine
- Record Identifier
- 9984065384002771
Metrics
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