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

• Main Authors: Naoko Shiba (Author), Xiao Yang (Author), Mitsuto Sato (Author), Shin Kadota (Author), Yota Suzuki (Author), Masahiro Agata (Author), Kohei Nagamine (Author), Masaki Izumi (Author), Yusuke Honda (Author), Tomoya Koganehira (Author), Hideki Kobayashi (Author), Hajime Ichimura (Author), Shinichiro Chuma (Author), Junichi Nakai (Author), Shugo Tohyama (Author), Keiichi Fukuda (Author), Daigo Miyazaki (Author), Akinori Nakamura (Author), Yuji Shiba (Author)
• Format: Book
• Published: Elsevier, 2023-12-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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,...