Target intelligence / Profile preview

MicroRNA 34a (miR-34a)

Target
miR-34a
Molecular classification
microRNA, Non-coding RNA, Regulatory RNA, Other
01

Overview

MicroRNA 34a (miR-34a) is a small, non-coding RNA encoded by the MIR34A gene in humans. It functions primarily as a post-transcriptional regulator of gene expression by binding to target mRNAs and leading to their degradation or inhibition of translation. miR-34a acts as a key tumor suppressor, directly or indirectly inhibiting genes involved in cell proliferation, cell cycle progression, survival, migration, metastasis, and epithelial-mesenchymal transition (EMT). Its expression is tightly regulated by the tumor suppressor protein p53 and is frequently downregulated in multiple cancers due to deletions, mutations, or epigenetic silencing. In addition to its role in oncology, miR-34a is implicated in fibrotic diseases, regulation of DNA repair, cell death, and senescence. As a therapeutic target, miR-34a mimics have been tested in clinical trials (notably MRX34), but widespread application is limited by safety and delivery challenges. Its altered expression serves as a biomarker for cancer diagnosis, prognosis, and possibly for monitoring anti-fibrotic responses[1][2][3][4][5][6][7][8].

Other names
MIR34AMIRN34AmiRNA34Amir-34mir-34amicroRNA 34aMIRN34 microRNA
02

Mechanism of action

Gene silencing via translational repression and mRNA destabilization of oncogenes and other targets; tumor suppression (by targeting genes in p53, TGF-β, NOTCH, PI3K/AKT pathways); regulation of apoptosis (targets SIRT1, BCL2, CD44, among others)

03

Biological functions

Post-transcriptional gene silencingCell cycle regulationCell proliferationApoptosisDiferentiationCell deathDNA repairRegulation of epithelial-mesenchymal transitionImmune responseSenescence
04

Disease associations

CancerFibrosisNeurodegenerative disease (suggested in some studies)Cardiovascular disease (suggested in some studies)Other
05

Safety considerations

Immune-related adverse reactions (noted in MRX34 trials)risk of off-target gene silencingdelivery and stability issues for RNA therapeutics
06

Interacting drugs

MRX34 (synthetic miR-34a mimic, previously in clinical trials; no longer active due to safety issues)
07

Biomarkers

miR-34a expression (biomarker for various cancers, metastasis, prognosis, and treatment response; investigational for fibrosis risk and other diseases)

Beyond the preview

Go deeper on MicroRNA 34a (miR-34a).

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 MicroRNA 34a (miR-34a).

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