Article

Depleting prion protein using splice-switching small molecules

2026-06-27

Abstract excerpt

Prion diseases are fatal neurodegenerative disorders driven by prion protein (PrP) misfolding, and lowering cellular PrP represents a promising therapeutic strategy. Here we report a small-molecule approach that reduces PrP by modulating pre-mRNA splicing of the PRNP gene. Through chemical modification of the clinically approved splicing modulator risdiplam, we generated CP3, the first class of compounds that sel...

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Literature Corpus work: 9016fc62-ef64-5c5c-b83e-57f8e7029e99
DOI: 10.64898/2026.06.26.734742

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