Target intelligence / Profile preview

Exosome component 2 (EXOSC2)

Target
EXOSC2
Molecular classification
Other (non-catalytic RNA exosome complex subunit, RNA-binding protein)
01

Overview

Exosome component 2 (EXOSC2) is a non-catalytic core subunit of the human RNA exosome complex, which is essential for 3'-to-5' exoribonucleolytic processing and degradation of various RNA species, including ribosomal RNA (rRNA), small nuclear RNA (snRNA), small nucleolar RNA (snoRNA), and aberrant messenger RNA (mRNA)[2][3][5]. The exosome consists of nine core subunits forming a cap and ring structure in eukaryotes; EXOSC2 (orthologous to yeast Rrp4) comprises part of the RNA-binding cap and stabilizes the core complex[3][5]. While itself enzymatically inactive, EXOSC2 is vital for the assembly, stability, and activity of the RNA exosome. Disruption of EXOSC2 destabilizes the complex and leads to severe cellular dysfunction, including defective RNA metabolism and reduced cell proliferation[2][3]. Genetic mutations in EXOSC2 cause rare syndromes with multisystem involvement, and its function is indispensable for cell survival[3].

Other names
Exosome complex component RRP4RRP4hRrp4pRrp4pSHRFp7Ribosomal RNA-processing protein 4homolog of yeast RRP43'-5'-exoribonuclease (RRP4)
02

Biological functions

RNA bindingRNA processing (maturation and degradation of rRNA, snRNA, snoRNA)RNA surveillance (elimination of aberrant mRNA, regulation of mRNA turnover)Cell proliferationRegulation of cell growth
03

Disease associations

Short stature, hearing loss, retinitis pigmentosa, and distinctive facies (rare genetic syndromes)Hepatocellular carcinoma (implicated, but not a direct driver)
04

Safety considerations

Mutations cause reduced cell proliferation and instability of the exosome complex.Pathogenic mutations lead to multisystem disorders such as SHRF (Short stature, Hearing loss, Retinitis pigmentosa, and Facies)Loss of function is lethal in model systems

Beyond the preview

Go deeper on Exosome component 2 (EXOSC2).

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 Exosome component 2 (EXOSC2).

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