Prevention of early-onset cardiomyopathy in Dmd exon 52–54 deletion mice by CRISPR-Cas9-mediated exon skipping

Prevention of early-onset cardiomyopathy in Dmd exon 52–54 deletion mice by CRISPR-Cas9-mediated exon skipping

Duchenne muscular dystrophy (DMD) is a disease with a life-threatening trajectory resulting from mutations in the dystrophin gene, leading to degeneration of skeletal muscle and fibrosis of cardiac muscle. The overwhelming majority of mutations are multiexonic deletions. We previously established a...

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Bibliographic Details
Main Authors: Matthew Rok, Tatianna Wai Ying Wong, Eleonora Maino, Abdalla Ahmed, Grace Yang, Elzbieta Hyatt, Kyle Lindsay, Sina Fatehi, Ryan Marks, Paul Delgado-Olguín, Evgueni A. Ivakine, Ronald D. Cohn
Format: Article
Language:English
Published: Elsevier 2023-09-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
CRISPR
Duchenne muscular dystrophy
exon skipping
genome editing
in vivo
cardiomyopathy
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050123001043

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http://www.sciencedirect.com/science/article/pii/S2329050123001043

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