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Lab Reports
July 3, 2013

Potential Targets Found for Neurodegenerative Disease

JAMA. 2013;310(1):22. doi:10.1001/jama.2013.7876

Using cross-species genetic screening in Drosophila, human cells, and a mouse model, researchers have identified proteins that modulate levels of the mutant ataxin 1 (ATXN1) protein that accumulates in the neurodegenerative disease spinocerebellar ataxia type 1 (SCA1). The proteins, components of the RAS-MAPK-MSK1 pathway, are potential therapeutic targets for the disorder, they said (Park J. Neuron. doi:10.1038/nature12204 [published online May 29, 2013]).

Because the pathway is involved in the early stages of SCA1, targeting it could potentially delay disease development. Indeed, drugs that inhibit different components in the pathway reduced ATXN1 levels and suppressed neurodegeneration in Drosophila and mice.