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Article

<i>DMD</i> antisense oligonucleotide mediated exon skipping efficiency is affected by flanking intron retention time and target position within the exon

2022-10-28

Abstract excerpt

Mutations in the DMD gene are causative for Duchenne muscular dystrophy (DMD). Antisense oligonucleotide (AON) mediated exon skipping to restore disrupted dystrophin reading frame is a therapeutic approach that allows production of a shorter but functional protein. As DMD causing mutations can affect most of the 78 exons encoding dystrophin, a wide variety of AONs are needed to treat the patient population. Desig...

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Identifiers and source

Literature Corpus work
742a1d65-4ef1-5f6a-bbb6-296908ae5377
DOI
10.1101/2022.10.28.514237
Open publication

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<i>DMD</i> antisense oligonucleotide mediated exon skipping efficiency is affected by flanking intron retention time and target position within the exonDOI 10.1101/2022.10.28.514237
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