IndraLab

Statements

ATXN3 inhibits mutated ATXN3. 3 / 3
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"Oral administration of the calpain inhibitor BDA-410 decreased both fragments formation and full-length ataxin-3 levels, reduced aggregation of mutant ataxin-3 and prevented cell injury and striatal and cerebellar degeneration."
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"Additionally, in a doxycycline-regulatable transgenic mouse model of SCA3, reducing production of mutant ATXN3 transcripts via doxycycline treatment beginning at 9weeks eliminated disease features."
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"Recently, treatment of a C. elegans model of SCA3 (spinocerebellar ataxia type 3; also known as Machado-Joseph disease) with 17-(allylamino)-17-demethoxygeldanamycin (17-AAG), an HSP90 inhibitor, successfully decreased the mutant ATXN3 aggregation and improved locomotor activity [XREF_BIBR]."
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