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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Detalles Bibliográficos
Autores principales: Rok, Matthew, Wong, Tatianna Wai Ying, Maino, Eleonora, Ahmed, Abdalla, Yang, Grace, Hyatt, Elzbieta, Lindsay, Kyle, Fatehi, Sina, Marks, Ryan, Delgado-Olguín, Paul, Ivakine, Evgueni A., Cohn, Ronald D.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10403712/
https://www.ncbi.nlm.nih.gov/pubmed/37545481
http://dx.doi.org/10.1016/j.omtm.2023.07.004

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