Article

SPLICER: A Highly Efficient Base Editing Toolbox That Enables <i>In Vivo</i> Therapeutic Exon Skipping

2024-04-02

Abstract excerpt

Exon skipping technologies enable exclusion of targeted exons from mature mRNA transcripts, which has broad applications in molecular biology, medicine, and biotechnology. Existing exon skipping techniques include antisense oligonucleotides, targetable nucleases, and base editors, which, while effective for specific applications at some target exons, remain hindered by shortcomings, including transient effects for...

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Literature Corpus work: f7656c46-aed4-5057-a778-4c9208de872d
DOI: 10.1101/2024.04.01.587650

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