RNAi Blocks Degeneration In Model Of Spinocerebellar Ataxia

NEW YORK (Reuters Health) - Findings from a murine study provide in vivo evidence that RNA interference (RNAi) is a useful therapy for dominant neurodegenerative disease. In a mouse model of spinocerebellar ataxia (SCA1), RNAi therapy suppressed the neurodegeneration that typically occurs.

"This is the first example of targeted gene silencing of a disease gene in the brains of live animals and it suggests that this approach may eventually be useful for human therapies," senior author Dr. Beverly Davidson, from the University of Iowa in Iowa City, said in a statement.

In the study, Dr. Davidson's team assessed the ability of RNAi to block the polyglutamine-induced neurotoxic effects of mutant ataxin-1 in mice with SCA1. Specifically, the animals were given an intracerebellar injection of adeno-associated virus vectors expressing short hairpin RNAs.

The researchers' findings appear in the July 4th online issue of Nature Medicine.

The intervention improved motor coordination in the test animals, the researchers note. Moreover, this therapy normalized cerebellar morphology and resolved the ataxin-1 inclusions found in Purkinje cells.

Our findings, the authors conclude, "have relevance to other polyglutamine-repeat disorders including Huntington disease and neurodegenerative disorders such as Alzheimer disease, in which inhibiting expression of a disease-linked protein would directly protect, or even reverse, disease phenotypes."

Source: Nat Med 2004. [ Google search on this article ]

MeSH Headings: Biological Sciences : Biology : Gene Expression Regulation : Genetics : Genetics, Biochemical : Molecular Biology : Spinocerebellar Ataxias : Gene Silencing : Biological Sciences

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