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.
Bonini
Nancy M
NM
Department of Biology and University of Pennsylvania, Philadelphia, Pennsylvania 19104, USA. nbonini@sas.upenn.edu
La Spada
Albert R
AR
D000818:Animals; D020868:Gene Silencing; D006801:Humans; D019636:Neurodegenerative Diseases / Q000235:genetics* / Q000628:therapy*; D034741:RNA, Small Interfering / Q000627:therapeutic use*
D034741:RNA, Small Interfering