Transcription profiling of mouse spinocerebellar ataxia type 3 (SCA3) transgenic and wild type animals treated with CCI-779 or placebo

ABSTRACT: Spinocerebellar ataxia type 3 is a neurodegenerative disorder caused by the expansion of the polyglutamine repeat region within the ataxin-3 protein. The mutant protein forms intracellular aggregates in the brain. However, the cellular mechanisms causing toxicity are still poorly understood and there are currently no effective treatments. In this study we show that administration of a rapamycin ester, CCI-779, improves motor performance in a transgenic mouse model of SCA3. CCI-779 inhibits mammalian target of rapamycin (mTOR) and hence upregulates protein degradation by autophagy. CCI-779 reduces the number of aggregates seen in the brains of transgenic mice and decreases levels of cytosolic soluble mutant ataxin-3, while endogenous wild-type protein levels remain unaffected. CCI-779 is de

ORGANISM(S): Mus musculus

SUBMITTER: Michael Bonin 

PROVIDER: E-GEOD-17994 | biostudies-arrayexpress |

REPOSITORIES: biostudies-arrayexpress