DG9 boosts PMO nuclear uptake and exon skipping to restore dystrophic muscle and cardiac function

DG9 boosts PMO nuclear uptake and exon skipping to restore dystrophic muscle and cardiac function

Abstract Duchenne muscular dystrophy (DMD) is a severe neuromuscular disorder caused by DMD gene mutations, leading to the loss of functional dystrophin. While antisense oligonucleotide (ASO)-mediated exon skipping offers therapeutic potential, its efficacy in cardiac muscle remains limited. Here, w...

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Main Authors: Md Nur Ahad Shah, Harry Wilton-Clark, Farhia Haque, Brooklynn Powell, Laura Edellein Sutanto, Radha Maradiya, Pavel Zhabyeyev, Rohini Roy Roshmi, Saeed Anwar, Tejal Aslesh, Kenji Rowel Q. Lim, Rika Maruyama, Anne Bigot, Courtney S. Young, Scott Bittner, Melissa J. Spencer, Hong M. Moulton, Gavin Y. Oudit, Toshifumi Yokota
Format: Article
Language:English
Published: Nature Portfolio 2025-05-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-025-59494-8
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https://doi.org/10.1038/s41467-025-59494-8

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