Article

Antisense oligonucleotide-based drug development for Cystic Fibrosis patients carrying the 3849+10kb C-to-T splicing mutation

2021-02-14

Abstract excerpt

Antisense oligonucleotide (ASO)-based drugs for splicing modulation were recently been approved for various genetic diseases with unmet need. Here we aimed to develop an ASO-based splicing modulation therapy for Cystic Fibrosis (CF) patients carrying the 3849+10kb C-to-T splicing mutation in the CFTR gene. We have screened, in FRT cells expressing this mutation, ~30 ASOs chemically modified with 2′-O-Methyl on a p...

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Literature Corpus work: 90bd64ce-5ce5-595c-9fb2-1356a6300053
DOI: 10.1101/2021.02.14.431123

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