Target intelligence / Profile preview

MAPT antisense RNA 1 (MAPT-AS1)

Target
MAPT-AS1
Molecular classification
Long non-coding RNA (lncRNA), Natural antisense transcript (NAT), Promoter-associated RNA, Other (non-protein coding RNA)
01

Overview

MAPT antisense RNA 1 (MAPT-AS1) is a natural antisense transcript (NAT) and long non-coding RNA expressed from the MAPT gene locus, which encodes microtubule-associated protein Tau. MAPT-AS1 is present in human neuronal tissues, including the brain, and has been investigated for its ability to modulate MAPT gene expression via epigenetic mechanisms such as promoter activity and DNA methylation. Initial studies suggested that MAPT-AS1 may repress MAPT expression and impact alternative splicing through regulation of promoter methylation and activity. However, more recent studies using multiple cell models, including human neurons, report that modulation of MAPT-AS1 levels does not alter MAPT mRNA or Tau protein expression, challenging its functional significance as a therapeutic target for tauopathies, including Alzheimer’s disease. There is some evidence of a role as a disease biomarker in neurodegenerative conditions and potential relevance in cancer biology, but its clinical utility remains unproven. MAPT-AS1 remains mainly a subject of basic and mechanistic research.

Other names
MAPT-ASMAPT antisense transcript 1MAPT natural antisense transcript
02

Mechanism of action

For experimental ASOs/siRNAs, the intended mechanism is knockdown of MAPT-AS1 transcript to modulate MAPT gene expression; however, recent studies report no consistent effect on MAPT mRNA or Tau protein in human neuronal models.

03

Biological functions

Epigenetic regulation (proposed)Regulation of gene expression (MAPT locus)Promoter activity modulation (MAPT gene)Other (possible trans-acting regulatory RNA)
04

Disease associations

Neurodegenerative disease (Alzheimer’s disease, Parkinson’s disease)Potential role in cancer (as a prognostic marker and possible therapeutic target in some cancers, as indicated by isolated studies)Other (possible biomarker of disease state)
05

Safety considerations

None documented, as MAPT-AS1 is not a therapeutic target with clinically approved drugs. Potential challenges include poor understanding of physiological function and redundancy with other regulatory RNAs.
06

Interacting drugs

None currently known. MAPT-AS1 is being investigated in preclinical models using antisense oligonucleotides (ASOs) and siRNAs for functional modulation, but no clinical drugs directly target MAPT-AS1.
07

Biomarkers

Reduced expression of MAPT-AS1 may correlate with disease status in Parkinson’s disease, but utility as an actionable biomarker is not yet established.

Beyond the preview

Go deeper on MAPT antisense RNA 1 (MAPT-AS1).

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 MAPT antisense RNA 1 (MAPT-AS1).

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