Connectivity mapping uncovers small molecules that modulate neurodegeneration in Huntington's disease models.

ABSTRACT:

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Huntington's disease (HD) is a genetic disease caused by a CAG trinucleotide repeat expansion encoding a polyglutamine tract in the huntingtin (HTT) protein, ultimately leading to neuronal loss and consequent cognitive decline and death. As no treatments for HD currently exist, several chemical screens have been performed using cell-based models of mutant HTT toxicity. These screens measured single disease-related endpoints, such as cell death, but had low 'hit rates' and limited dimensionality for therapeutic detection. Here, we have employed gene expression microarray analysis of HD samples--a snapshot of the expression of 25,000 genes--to define a gene expression signature for HD from publically available data. We used this information to mine a database for chemicals

SUBMITTER: Smalley JL 

PROVIDER: S-EPMC4762922 | biostudies-literature | 2016 Feb

REPOSITORIES: biostudies-literature