Article
Targeting the VCP-binding motif of ataxin-3 improves phenotypes in Drosophila models of Spinocerebellar Ataxia Type 3.
Neurobiology of disease - 1 Dec 2021
Johnson Sean L, Libohova Kozeta, Blount Jessica R, Sujkowski Alyson L, Prifti Matthew V, Tsou Wei-Ling, Todi Sokol V
Abstract excerpt
Of the family of polyglutamine (polyQ) neurodegenerative diseases, Spinocerebellar Ataxia Type 3 (SCA3) is the most common. Like other polyQ diseases, SCA3 stems from abnormal expansions in the CAG triplet repeat of its disease gene resulting in elongated polyQ repeats within its protein, ataxin-3. Various ataxin-3 protein domains contribute to its toxicity, including the valosin-containing protein (VCP)-binding...
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