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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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Detalles Bibliográficos
Autores principales:	Wang, Derek W., Mokhonova, Ekaterina I., Kendall, Genevieve C., Becerra, Diana, Naeini, Yalda B., Cantor, Rita M., Spencer, Melissa J., Nelson, Stanley F., Miceli, M. Carrie
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2018
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5992346/ https://www.ncbi.nlm.nih.gov/pubmed/29858053 http://dx.doi.org/10.1016/j.omtn.2018.02.002

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