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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Veröffentlicht in:Mol Ther Nucleic Acids
Hauptverfasser: 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
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Society of Gene & Cell Therapy 2018
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Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5992346/
https://ncbi.nlm.nih.gov/pubmed/29858053
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2018.02.002
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