Article

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

2025-04-30

Abstract excerpt

Frontotemporal dementia with parkinsonism linked to chromosome 17 (FTDP-17) is caused by the aberrant alternative pre-mRNA splicing of microtubule-associated protein tau ( MAPT ) exon 10, the inclusion of which encodes for a toxic tau protein harboring four microtube domains (4R tau). Here, we describe the design of an RNA-targeted small molecule that thermodynamically stabilizes the structure of a pre-mRNA splic...

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Literature Corpus work: 88c54f65-18c6-52b8-9fec-5323bb537790
DOI: 10.1101/2025.04.30.651234

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