Exon skipping induces uniform dystrophin rescue with dose-dependent restoration of serum miRNA biomarkers and muscle biophysical properties

Therapies that restore dystrophin expression are presumed to correct Duchenne muscular dystrophy (DMD), with antisense-mediated exon skipping being the leading approach. Here we aimed to determine whether exon skipping using a peptide-phosphorodiamidate morpholino oligonucleotide (PPMO) conjugate re...

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Detalles Bibliográficos
Autores principales: Chwalenia, Katarzyna, Oieni, Jacopo, Zemła, Joanna, Lekka, Małgorzata, Ahlskog, Nina, Coenen-Stass, Anna M.L., McClorey, Graham, Wood, Matthew J.A., Lomonosova, Yulia, Roberts, Thomas C.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9464767/
https://www.ncbi.nlm.nih.gov/pubmed/36159597
http://dx.doi.org/10.1016/j.omtn.2022.08.033

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9464767/
https://www.ncbi.nlm.nih.gov/pubmed/36159597
http://dx.doi.org/10.1016/j.omtn.2022.08.033

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