Long-Term Efficacy of Systemic Multiexon Skipping Targeting Dystrophin Exons 45–55 With a Cocktail of Vivo-Morpholinos in Mdx52 Mice

Antisense-mediated exon skipping, which can restore the reading frame, is a most promising therapeutic approach for Duchenne muscular dystrophy. Remaining challenges include the limited applicability to patients and unclear function of truncated dystrophin proteins. Multiexon skipping targeting exon...

Ausführliche Beschreibung

Gespeichert in:
Bibliographische Detailangaben
Veröffentlicht in:Mol Ther Nucleic Acids
Hauptverfasser: Echigoya, Yusuke, Aoki, Yoshitsugu, Miskew, Bailey, Panesar, Dharminder, Touznik, Aleksander, Nagata, Tetsuya, Tanihata, Jun, Nakamura, Akinori, Nagaraju, Kanneboyina, Yokota, Toshifumi
Format: Artigo
Sprache:Inglês
Veröffentlicht: Nature Publishing Group 2015
Schlagworte:
Original Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4345310/
https://ncbi.nlm.nih.gov/pubmed/25647512
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2014.76
Tags: Tag hinzufügen
Keine Tags, Fügen Sie den ersten Tag hinzu!

Ähnliche Einträge