Target intelligence / Profile preview

Ataxin-1 (ATXN1)

Target: ATXN1
Molecular classification: Transcription factor, RNA-binding protein, Other

Overview

Ataxin-1 is a nuclear protein involved in transcriptional regulation and RNA metabolism. It plays a crucial role in the pathogenesis of spinocerebellar ataxia type 1 (SCA1), a neurodegenerative disorder caused by the expansion of a polyglutamine tract within the protein. The normal function of Ataxin-1 involves interactions with various proteins, including Capicua (CIC) and splicing factors. Dysregulation of Ataxin-1 has also been implicated in cancer, Alzheimer's disease, and multiple sclerosis, highlighting its complex role in cellular processes.

Other names: Spinocerebellar ataxia type 1 proteinSCA1 ProteinAtxn-1 Protein

Biological functions

Regulation of gene expressionChromatin bindingRNA metabolismImmunomodulation

Disease associations

Spinocerebellar ataxia type 1 (SCA1)Cancer (risk modulation)Alzheimer’s disease (risk)Multiple sclerosis (susceptibility)

Safety considerations

Potential for off-target effects due to broad role in gene regulationChallenge of selectively targeting mutant Ataxin-1 without affecting wild-type functionNeurotoxicityLoss of function mutations may be associated with cancer development, Alzheimer's disease pathogenesis, and susceptibility loci for multiple sclerosis.

Biomarkers

Polyglutamine tract length (CAG repeats) in ATXN1 gene

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