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Updated: Jul 12, 2026

Exon Skipping in Directly Reprogrammed Myotubes Obtained from Human Urine-Derived Cells
Published on: May 7, 2020
Rewriting Duchenne muscular dystrophy therapy
Paloma Gonzalez-Perez1, Craig Blackstone1
1Department of Neurology, Mass General Brigham and Harvard Medical School, Boston, MA 02114, USA.
Abstract:
In this issue of Cell, Guo et al. report the development of a new exon-skipping, RNA-editing-based therapy for Duchenne muscular dystrophy. The dual mechanism of action through both ADAR-dependent and -independent pathways has the potential to be more effective and require a lower dosing frequency than currently available ASO-based exon-skipping treatments.
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