MOTS‐c promotes phosphorodiamidate morpholino oligomer uptake and efficacy in dystrophic mice

Antisense oligonucleotide (AO)‐mediated exon‐skipping therapies show promise in Duchenne muscular dystrophy (DMD), a devastating muscular disease caused by frame‐disrupting mutations in the DMD gene. However, insufficient systemic delivery remains a hurdle to clinical deployment. Here, we demonstrat...

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Udgivet i:EMBO Mol Med
Main Authors: Ran, Ning, Lin, Caorui, Leng, Ling, Han, Gang, Geng, Mengyuan, Wu, Yingjie, Bittner, Scott, Moulton, Hong M, Yin, HaiFang
Format: Artigo
Sprog:Inglês
Udgivet: John Wiley and Sons Inc. 2020
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Articles
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7863382/
https://ncbi.nlm.nih.gov/pubmed/33337582
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.15252/emmm.202012993
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