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Article

Design and evaluation of locked nucleic acid-based splice-switching oligonucleotidesin vitro

16 Jun 2014

Abstract excerpt

Antisense-mediated modulation of pre-mRNA splicing is an attractive therapeutic strategy for genetic diseases. Currently, there are few examples of modulation of pre-mRNA splicing using locked nucleic acid (LNA) antisense oligonucleotides, and, in particular, no systematic study has addressed the optimal design of LNA-based splice-switching oligonucleotides (LNA SSOs). Here, we designed a series of LNA SSOs...

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PMID: 24935206
DOI: 10.1093/nar/gku512

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