Target intelligence / Profile preview

Microtubule-associated protein tau pre-mRNA (MAPT pre-mRNA)

Target
MAPT pre-mRNA
Molecular classification
Other (pre-mRNA; not a protein, receptor, enzyme, transporter, or classical signaling molecule. It is a gene transcript.)
01

Overview

The **microtubule-associated protein tau pre-mRNA (MAPT pre-mRNA)** is the precursor messenger RNA transcript encoded by the MAPT gene, responsible for producing tau protein isoforms by alternative splicing. The structure of tau pre-mRNA, particularly at specific exon-intron junctions and within the 5′ and 3′ untranslated regions, plays a key role in regulating exon inclusion (notably exon 10), which determines the balance of tau isoforms expressed in neurons[1][2][4][5]. Disease-associated mutations that alter the secondary structure of tau pre-mRNA can disrupt normal splicing patterns, producing pathogenic tau isoforms linked to neurodegenerative diseases such as Alzheimer’s disease and frontotemporal dementia (“tauopathies”)[1][2][4][5]. Small molecules (e.g., mitoxantrone) have been identified that specifically bind to and stabilize key secondary structures within tau pre-mRNA, thereby altering splicing and representing a promising therapeutic approach to treat tauopathies[4]. The identification of stable and highly conserved structured regions throughout tau pre-mRNA underscores its relevance as a therapeutic target for modulation of tau gene expression and protein isoform production[1][2].

Other names
tau pre-mRNAMAPT pre-mRNAmicrotubule-associated protein tau precursor mRNA
02

Mechanism of action

Small molecules stabilizing pathological RNA secondary structure (e.g., stem-loop) to influence tau exon 10 splicing and isoform production[4] Therapeutics targeting splicing regulatory elements in pre-mRNA[4]

03

Biological functions

Regulation of alternative splicingGene expression regulationmRNA stability
04

Disease associations

Neurodegenerative disease (especially tauopathies such as Alzheimer’s disease and frontotemporal dementia)Other (Aberrant splicing/mutation linked to inherited neurodegeneration)
05

Safety considerations

Off-target effects of RNA-binding drugsUnintended modulation of splicing in other genes (general concern for RNA-directed therapies)Potential impact on non-neuronal MAPT expression
06

Interacting drugs

Mitoxantrone (binds and stabilizes tau pre-mRNA secondary structure)
07

Biomarkers

Mutations in MAPT exon 10-intron 10 segment (e.g., G(+3)A, C(+14)T)[1][2][5]MAPT alternative splicing patterns/expression (e.g., exon 10 inclusion/exclusion)

Beyond the preview

Go deeper on Microtubule-associated protein tau pre-mRNA (MAPT pre-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 Microtubule-associated protein tau pre-mRNA (MAPT pre-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