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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Detalles Bibliográficos
Autores principales: Goossens, Remko, Verwey, Nisha, Ariyurek, Yavuz, Schnell, Fred, Aartsma-Rus, Annemieke
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Taylor & Francis 2023
Materias:
Research Paper
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10481881/
https://www.ncbi.nlm.nih.gov/pubmed/37667454
http://dx.doi.org/10.1080/15476286.2023.2254041

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10481881/
https://www.ncbi.nlm.nih.gov/pubmed/37667454
http://dx.doi.org/10.1080/15476286.2023.2254041

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