Golodirsen restores DMD transcript imbalance in Duchenne Muscular Dystrophy patient muscle cells

Golodirsen restores DMD transcript imbalance in Duchenne Muscular Dystrophy patient muscle cells

Abstract Background Antisense oligonucleotides (AON) represent a promising treatment for Duchenne muscular dystrophy (DMD) carrying out-of-frame deletions, but also show limitations. In a completed clinical trial golodirsen, approved by FDA to induce skipping of DMD gene exon 53 in eligible patients...

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Bibliographic Details
Main Authors: Rachele Rossi, Silvia Torelli, Marc Moore, Pierpaolo Ala, Jennifer Morgan, Jyoti Malhotra, Francesco Muntoni
Format: Article
Language:English
Published: BMC 2024-11-01
Series:Skeletal Muscle
Subjects:
Antisense oligonucleotide
Duchenne muscular dystrophy
Golodirsen
Morpholino
Transcript imbalance.
Online Access:https://doi.org/10.1186/s13395-024-00360-4
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