Article

Divalent siRNA for prion disease

2024-12-09

Abstract excerpt

Prion protein (PrP) lowering is effective in animal models of prion disease and is being tested clinically in prion disease patients, but there remains a need for more potent PrP-lowering drug candidates. Inspired by the reported potency and duration of action of divalent short interfering RNA (siRNA), a new oligonucleotide drug modality for the central nervous system, we sought to discover and develop a new PrP-l...

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Literature Corpus work: 68c705cd-4e83-5b4b-a05f-0e098af52669
DOI: 10.1101/2024.12.05.627039

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