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

doi:10.1126/scitranslmed.ady6759

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Article

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

Science translational medicine - 19 Aug 2026

Silva M Catarina, Lindmeier Hannah, Pigini Paolo, Laughlin Jennifer, Yu Yong, Morrill Christie, Arnold Michael A, Barraza Scott J, Saadipour Khalil, Dieterich Monal, Minnella Angela, Wang Lin-Ing, Li Wencheng, Datta Kausiki, Zhang Nanjing, Narasimhan Jana, Trotta Christopher R, Woll Matthew G, Welch Ellen M, Benzow Kellie, Karanjeet Kul, Lotz Steven, Bertucci Taylor, Temple Sally, Haggarty Stephen J, Koob Michael, Trimmer Jeffrey, Weetall Marla, Morini Elisabetta

Abstract excerpt

Tauopathies are neurodegenerative diseases characterized by the pathological 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. Exon 10 and adjacent introns of the MAPT gene are a hotspot for pathogenic variants,...

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PMID: 42616837
DOI: 10.1126/scitranslmed.ady6759

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