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

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

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 partially...

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Bibliographic Details
Main Authors: Derek W. Wang, Ekaterina I. Mokhonova, Genevieve C. Kendall, Diana Becerra, Yalda B. Naeini, Rita M. Cantor, Melissa J. Spencer, Stanley F. Nelson, M. Carrie Miceli
Format: Article
Language:English
Published: Elsevier 2018-06-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
exon skipping
Duchenne muscular dystrophy
dystrophin
dantrolene
combination therapy
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253118300167

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