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...
| Auteurs principaux: | , , , , |
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| Format: | Article |
| Langue: | English |
| Publié: |
Taylor & Francis Group
2023-12-01
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| Collection: | RNA Biology |
| Sujets: | |
| Accès en ligne: | http://dx.doi.org/10.1080/15476286.2023.2254041 |