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Ataxin-3 (ATXN3)
- Target
- ATXN3
- Molecular classification
- Deubiquitinating enzyme, DUB
01
Overview
Ataxin-3 is a deubiquitinating enzyme (DUB) encoded by the *ATXN3* gene. It plays a role in protein quality control through its DUB activity and interaction with the ubiquitin-proteasome system. Expansion of a polyglutamine (polyQ) repeat region in ataxin-3 leads to its aggregation and is the underlying cause of spinocerebellar ataxia type 3 (SCA3), also known as Machado-Joseph disease.
- Other names
- Machado-Joseph disease protein 1MJD1
02
Mechanism of action
Inhibition of deubiquitination activity; Modulation of protein aggregation
03
Biological functions
DeubiquitinationProtein quality controlUbiquitin-proteasome system regulation
04
Disease associations
Spinocerebellar ataxia type 3Machado-Joseph diseaseNeurodegenerative disease
05
Safety considerations
Potential for off-target effects due to DUB activityChallenges in targeting protein aggregates
06
Biomarkers
Ataxin-3 protein levelsPolyglutamine repeat length
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