Repurposing Dantrolene for Long-Term Combination Therapy to Potentiate Antisense-Mediated DMD Exon Skipping in the mdx Mouse

• Main Authors: Derek W. Wang (Author), Ekaterina I. Mokhonova (Author), Genevieve C. Kendall (Author), Diana Becerra (Author), Yalda B. Naeini (Author), Rita M. Cantor (Author), Melissa J. Spencer (Author), Stanley F. Nelson (Author), M. Carrie Miceli (Author)
• Format: Book
• Published: Elsevier, 2018-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Duchenne muscular dystrophy (DMD) is caused by mutations in DMD, resulting in loss of dystrophin, which is essential to muscle health. DMD "exon skipping" uses anti-sense oligo-nucleotides (AONs) to force specific exon exclusion during mRNA processing to restore reading frame and rescue of...