Article

Modulation of prion protein expression through cryptic splice site manipulation

2023-12-19

Abstract excerpt

Lowering expression of prion protein (PrP) is a well-validated therapeutic strategy in prion disease, but additional modalities are urgently needed. In other diseases, small molecules have proven capable of modulating pre-mRNA splicing, sometimes by forcing inclusion of cryptic exons that reduce gene expression. Here, we characterize a cryptic exon located in human PRNP ’s sole intron and evaluate its potential t...

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Literature Corpus work: bbf0c44b-8bd7-5a4f-9f50-6c3940683433
DOI: 10.1101/2023.12.19.572439

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