Article

<i>MAPT</i> Splicing Modulators as a Therapeutic Strategy for Tauopathies

2025-05-10

Abstract excerpt

<h4>ABSTRACT</h4> Tauopathies are neurodegenerative diseases characterized by the abnormal accumulation of microtubule-associated protein tau (MAPT) in the brain. These disorders, like frontotemporal dementia (FTD-Tau), currently lack effective therapies and can occur sporadically or be inherited when associated with MAPT gene mutations. The MAPT gene region encompassing exon 10 and adjacent introns is a hotspo...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: 0886f286-b94b-57da-a37a-5692b20cc676
DOI: 10.1101/2025.05.06.652428

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

<i>MAPT</i> Splicing Modulators as a Therapeutic Strategy for TauopathiesDOI 10.1101/2025.05.06.652428Read

MAPT splicing modulators reduce 4R tau and rescue tauopathy phenotypes in human neurons and in a mouse model.Semantic proximity Read

Dysregulated coordination of <i>MAPT</i> exon 2 and exon 10 splicing underlies different tau pathologies in PSP and ADSemantic proximity Read

Design of an orally bioavailable small molecule that modulates the microtubule-associated protein tau’s pre-mRNA splicingSemantic proximity Read

Alternative splicing of exon 10 in the tau gene as a target for treatment of tauopathies.Semantic proximity Read

Human iPSC 4R tauopathy model uncovers modifiers of tau propagationSemantic proximity Read

HCS-Splice: A High-Content Screening Method to Advance the Discovery of RNA Splicing-Modulating TherapeuticsSemantic proximity Read

HCS-Splice: A High-Content Screening Method to Advance the Discovery of RNA Splicing-Modulating Therapeutics.Semantic proximity Read

<i>HnRNPAB</i> -targeted antisense oligonucleotides ameliorate tau pathology and cognitive deficits by modulating Alzheimer’s disease-associated alternative splicingSemantic proximity Read

Human iPSC 4R tauopathy model uncovers modifiers of tau propagation.Semantic proximity Read

Scalable human neuronal models of tauopathy producing endogenous seed-competent 4R tauSemantic proximity Read

Tauopathies: Deciphering Disease Mechanisms to Develop Effective Therapies.Semantic proximity Read

Bottom-up design of small molecules that stimulate exon 10 skipping in mutant MAPT pre-mRNA.Semantic proximity Read