Target intelligence / Profile preview

microRNA 128-1 (miR-128-1)

Target
miR-128-1
Molecular classification
microRNA, non-coding RNA, post-transcriptional gene regulator
01

Overview

MicroRNA 128-1 is a small non-coding RNA originally identified as a brain-enriched miRNA, with strong expression in differentiated neuronal cells and a key role in nervous system development and function[2]. It is one of two genes (miR-128-1 and miR-128-2) that generate the mature miR-128 form, with miR-128-1 located within the intronic region of the R3HDM1 gene on chromosome 2q21.3. miR-128-1 regulates oncogenic and stem cell factors such as BMI-1, NANOG, and TGFBR1, acting as a tumor suppressor in glioblastoma, prostate, and lung cancer by inhibiting cell proliferation, self-renewal, and angiogenesis[1][2]. Its tumor suppressive function is executed through direct downregulation of these target genes, leading to reduced cancer stem cell maintenance and tumor growth. Aberrant expression of miR-128-1—whether downregulation in solid tumors or context-dependent upregulation—has been linked to cancer aggressiveness, therapy resistance, and altered prognosis[1][2]. Restoration or inhibition of miR-128-1 is under preclinical investigation as a therapeutic strategy in multiple malignancies. No small-molecule drugs targeting miR-128-1 exist at this time, but gene therapy approaches leveraging synthetic miRNA mimics or antisense technologies are in experimental development[2].

Other names
hsa-mir-128ahsa-mir-128-1mir-128-1MIR128AMIRN128-1MIRN128AmicroRNA 128aMIR128-1
02

Mechanism of action

miR-128-1 functions by binding to complementary sequences in target mRNAs, leading to mRNA degradation or inhibition of translation (post-transcriptional repression). Candidate therapeutic mechanisms include miR-128-1 mimicry to downregulate oncogene expression, or inhibition to reverse tumor suppressor effects in settings where its activity is deleterious.

03

Biological functions

Regulation of stem cell propertiesNeuronal differentiation and functionCell proliferationApoptosisTumor suppressionRegulation of oncogene and tumor suppressor gene expression
04

Disease associations

Cancer (tumor suppressor role in glioblastoma, prostate cancer, neuroblastoma, lung cancer)Neurodevelopmental disordersPotential roles in cardiovascular and metabolic diseases (based on broad regulatory function)
05

Safety considerations

Potential off-target effects due to miRNA’s ability to regulate multiple mRNA targetsRisks of disrupting normal neuronal or regenerative functions, as miR-128-1 also controls neural differentiation and maintenanceDelivery and stability of miRNA-based therapeutics in clinical settings
06

Interacting drugs

miRNA mimics

1 more in the full profile.

07

Biomarkers

Low miR-128-1 expression (potential biomarker for poor prognosis in certain cancers: prostate, glioblastoma, lung cancer)Target genes (e.g., BMI-1, NANOG, TGFBR1, VEGF-C, TERT, NEK2)

Beyond the preview

Go deeper on microRNA 128-1 (miR-128-1).

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 128-1 (miR-128-1).

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