Silencing polyglutamine degeneration with RNAi.
Nine dominantly inherited neurodegenerative diseases are caused by expansion of a CAG repeat encoding glutamine. An important development in the study of such "polyglutamine" diseases was the realization that merely shutting off expression of a disease-encoding transgene could arrest progression in animal models with significant disease pathology. Such studies opened the door to a powerful new therapeutic approach now being pioneered: silencing of the dominant disease allele by RNA-mediated interference (RNAi), for the arrest--and potential reversal--of the disease process.[1]References
- Silencing polyglutamine degeneration with RNAi. Bonini, N.M., La Spada, A.R. Neuron (2005) [Pubmed]
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