Systematic evaluation of 2′-Fluoro modified chimeric antisense oligonucleotide-mediated exon skipping in vitro
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DOI: 10.1038/s41598-019-42523-0
Abstract: Antisense oligonucleotide (AO)-mediated splice modulation has been established as a therapeutic approach for tackling genetic diseases. Recently, Exondys51, a drug that aims to correct splicing defects in the dystrophin gene was approved by the US… read more here.
Keywords: chemistry; systematic evaluation; antisense oligonucleotide; oligonucleotide mediated ... See more keywords