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

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

Abstract 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 d...

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Bibliographic Details
Main Authors: Ning Ran, Caorui Lin, Ling Leng, Gang Han, Mengyuan Geng, Yingjie Wu, Scott Bittner, Hong M Moulton, HaiFang Yin
Format: Article
Language:English
Published: Springer Nature 2021-02-01
Series:EMBO Molecular Medicine
Subjects:
duchenne muscular dystrophy
energy
exon‐skipping
MOTS‐c
PMO
Online Access:https://doi.org/10.15252/emmm.202012993

Internet

https://doi.org/10.15252/emmm.202012993

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