Identification of compounds that modulate ataxin-3 aggregation and neurological dysfunction in a C. elegans model of Machado-Joseph disease.(Research Grant Award)(Report)(Brief article)

Machado-Joseph disease (MJD), also known as spinocerebellar ataxia type 3, is caused by mutations in the protein ataxin-3 that make this protein prone to aggregate and toxic to specific groups of neurons. This leads to the progressive neurodegeneration and to the typical symptoms of MJD: ataxia and severe limitations in eye movements among others. Currently there is no effective treatment for this disorder. Our goal was to test a large number of commercially available chemical compounds as potential therapies for MJD. For this, we used a model of the disease in the worm C. elegans, which in spite of being a very simple animal possesses a very well characterized nervous system, is quite …