Target intelligence / Profile preview

Ataxin-3 mRNA (ATXN3 mRNA)

Target
ATXN3 mRNA
Molecular classification
mRNA, Transcript
01

Overview

Ataxin-3 mRNA encodes the ataxin-3 protein, a deubiquitinating enzyme (DUB) involved in various cellular processes. The gene is known as ATXN3 and is most notably associated with the neurodegenerative disorder Machado–Joseph disease (MJD), also called spinocerebellar ataxia type 3 (SCA3). The pathogenic role arises primarily when mutations/expansions occur within this mRNA’s coding sequence leading to altered protein products that aggregate abnormally.

Other names
ATXN3 transcriptMachado-Joseph disease protein 3 mRNASCA3 mRNA
02

Mechanism of action

N/A

03

Biological functions

Encoding of deubiquitinating enzymeRegulation of protein homeostasis (indirect)Regulation of transcription (indirect)Maintenance of genomic stability (indirect)
04

Disease associations

Machado-Joseph disease/SCA3 (indirect)Neurodegenerative disease (indirect)Testicular cancer (indirect)Breast cancer (indirect)Gastric cancer (indirect)Thyroid cancer (indirect)
05

Safety considerations

N/A - mRNA itself does not pose direct safety concerns, but its encoded protein does when mutated.
06

Biomarkers

ATXN3 mRNA levels (potential)CAG repeat length in ATXN3 mRNA

Beyond the preview

Go deeper on Ataxin-3 mRNA (ATXN3 mRNA).

Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.

Drug pipeline

Full profile access

Explore the programs pursuing this target and their development progress.

  • Drug candidates
  • Developers
  • Development stage

Clinical trials

Full profile access

Follow the clinical studies evaluating therapies directed at this target.

  • Trial design
  • Status
  • Readouts

Competitive landscape

Full profile access

Compare approaches across drug candidates, modalities, and indications.

  • Programs
  • Modalities
  • Indications

Literature & evidence

Full profile access

Investigate the research and source evidence behind target biology and development.

  • Publications
  • Sources
  • Analysis

Patents

Full profile access

Explore patent activity around therapies and technologies addressing this target.

  • Patents
  • Assignees
  • Technologies

Research & analysis

Full profile access

Connect target biology, drug development, and emerging evidence in your research.

  • Biology
  • Development news
  • Analysis

Bring the full picture into focus.

See how Gosset can support your research on Ataxin-3 mRNA (ATXN3 mRNA).

Explore the full profile

Gosset Free

Get started with Gosset.

Enter your work email and we’ll be in touch with next steps.

Work email preferred.

Book a call