Target intelligence / Profile preview

Atrophin 1 (ATN1)

Target: ATN1
Molecular classification: Transcription factor, Transcriptional corepressor

Overview

Atrophin 1 (ATN1) is a protein involved in transcriptional repression, primarily in neurons. It contains a CAG trinucleotide repeat region, the expansion of which causes dentatorubral-pallidoluysian atrophy (DRPLA), a neurodegenerative disorder. ATN1 interacts with several nuclear proteins, and participates in signaling pathways. It acts as a transcriptional corepressor and plays a role in cell polarity and transcription control in progenitor cells.

Other names: dentatorubral-pallidoluysian atrophy proteinDRPLAD12S755EB37NOD[1][4]

Biological functions

Transcriptional repressionRegulation of cell polarityRegulation of transcription controlSignal transduction

Disease associations

Neurodegenerative diseaseGenetic disorder

Safety considerations

Targeting transcriptional regulators can have broad effects.Development of therapeutics for neurodegenerative diseases is challenging.

Biomarkers

CAG repeat length in ATN1 (for DRPLA diagnosis and risk assessment)

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