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Related Experiment Video

Updated: Sep 15, 2025

Modulation of Tau Subcellular Localization as a Tool to Investigate the Expression of Disease-related Genes
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MAPT Splicing Modulators as a Therapeutic Strategy for Tauopathies.

M Catarina Silva, Hannah Lindmeier, Paolo Pigini

    Biorxiv : the Preprint Server for Biology
    |July 14, 2025
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    Summary

    Researchers discovered novel splicing modulator compounds (SMCs) that target the MAPT gene. These compounds reduce harmful 4R tau protein, a hallmark of tauopathies, and show therapeutic potential for neurodegenerative diseases.

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    Area of Science:

    • Neuroscience
    • Genetics
    • Pharmacology

    Background:

    • Tauopathies are neurodegenerative diseases defined by abnormal tau protein accumulation.
    • Current therapies for tauopathies, such as frontotemporal dementia (FTD-Tau), are limited.
    • Pathogenic variants in the MAPT gene, particularly affecting exon 10 splicing, drive 4R tauopathies.

    Purpose of the Study:

    • To discover and characterize novel splicing modulator compounds (SMCs) targeting MAPT pre-mRNA splicing.
    • To evaluate the therapeutic efficacy of SMCs in preclinical models of 4R tauopathies.

    Main Methods:

    • Identification of novel SMCs promoting MAPT exon 10 exclusion.
    • Testing SMC efficacy in FTD patient-derived neuronal models with P301L or S305N mutations.
    • Assessment of SMC effects on 4R tau expression, tau phosphorylation, and aggregation.
    • In vivo validation in a gene-replacement mouse model with the tau-N279K splicing mutation.

    Main Results:

    • SMCs successfully promoted MAPT exon 10 exclusion.
    • Treatment reduced 4R tau expression, hyperphosphorylated tau (pTau), and insoluble tau aggregates.
    • SMC treatment rescued tau-associated neuronal toxicity in patient-derived models.
    • Lead SMC corrected the 3R/4R splice ratio in vivo and reduced brain pTau in a mouse model.

    Conclusions:

    • SMC-based modulation of MAPT pre-mRNA splicing is a viable therapeutic strategy for 4R tauopathies.
    • These novel compounds demonstrate significant potential for treating neurodegenerative conditions characterized by aberrant tau processing.