DMD antisense oligonucleotide mediated exon skipping efficiency correlates with flanking intron retention time and target position within the exon

DMD antisense oligonucleotide mediated exon skipping efficiency correlates with flanking intron retention time and target position within the exon

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 affec...

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Bibliographic Details
Main Authors: Remko Goossens, Nisha Verwey, Yavuz Ariyurek, Fred Schnell, Annemieke Aartsma-Rus
Format: Article
Language:English
Published: Taylor & Francis Group 2023-12-01
Series:RNA Biology
Subjects:
duchenne muscular dystrophy
dystrophin
dmd
antisense oligonucleotides
exon skipping
aon
aso
Online Access:http://dx.doi.org/10.1080/15476286.2023.2254041

Internet

http://dx.doi.org/10.1080/15476286.2023.2254041

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