IndraLab

Statements

ATXN3 inhibits mutated ATXN3. 5 / 5
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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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"This strategy has been used for spinocerebellar ataxia type 3 (SCA3) , where CRISPR/Cas9-mediated deletion of exon 10 including the CAG expansion in the ATXN3 gene effectively eliminates the aggregation of mutant ataxin-3 protein ."
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"We conclude that insertion of PAS upstream of expanded CAG repeats using CRISPR/Cas9 is a viable way to eliminate ataxin-3 aggregates and rescue the Golgi apparatus phenotype in SCA3.He et al. have shown that CRISPR/Cas9 genome editing technique in SCA3 patient-derived iPSCs can eliminate the production of mutant ataxin-3 protein and correct mitochondrial dysfunction by replacing 74 CAG repeats with 17 CAG repeats ."
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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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