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New Lead in Huntington's Disease Treatment

Molecular therapy in mouse model shows promise

THURSDAY, Oct. 11 (HealthDay News) -- In a mouse model for Huntington's disease, the small molecule C2-8 has been shown to cross the blood-brain barrier, leading to decreased aggregation of the mutant huntingtin protein and improved motor performance in mice, according to the results of a study published online Oct. 9 in the Proceedings of the National Academy of Sciences.

Vanita Chopra, Ph.D., of the Department of Neurology at Harvard Medical School in Charlestown, Mass., and colleagues used R6/2 transgenic mice to examine whether C2-8 has potential for further exploration as a therapy for Huntington's disease.

Treated mice had less neuronal atrophy and smaller huntingtin aggregates, and the effects were seen at non-toxic doses. The authors note that this is the first drug-like, brain-penetrable aggregation inhibitor that has been found from cell-based, high-throughput screens for the reduction of huntingtin aggregation.

"C2-8 provides an essential tool to help elucidate mechanisms of neurodegeneration in Huntington's disease and a therapeutic lead for further optimization and development," the authors conclude.

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