Efficacy of exon-skipping therapy for DMD cardiomyopathy with mutations in actin binding domain 1

Exon-skipping therapy is a promising treatment strategy for Duchenne muscular dystrophy (DMD), which is caused by loss-of-function mutations in the DMD gene encoding dystrophin, leading to progressive cardiomyopathy. In-frame deletion of exons 3–9 (Δ3–9), manifesting a very mild clinical phenotype,...

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Detalles Bibliográficos
Autores principales: Shiba, Naoko, Yang, Xiao, Sato, Mitsuto, Kadota, Shin, Suzuki, Yota, Agata, Masahiro, Nagamine, Kohei, Izumi, Masaki, Honda, Yusuke, Koganehira, Tomoya, Kobayashi, Hideki, Ichimura, Hajime, Chuma, Shinichiro, Nakai, Junichi, Tohyama, Shugo, Fukuda, Keiichi, Miyazaki, Daigo, Nakamura, Akinori, Shiba, Yuji
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/PMC10654596/
https://www.ncbi.nlm.nih.gov/pubmed/38028197
http://dx.doi.org/10.1016/j.omtn.2023.102060

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