In Vivo Evaluation of Multiple Exon Skipping with Peptide-PMOs in Cardiac and Skeletal Muscles in Dystrophic Dogs
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Publisher
Springer New York
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http://link.springer.com/content/pdf/10.1007/978-1-4939-8651-4_23
Reference50 articles.
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3. Flanigan KM (2014) Duchenne and Becker muscular dystrophies. Neurol Clin 32(3):671–688., viii. https://doi.org/10.1016/j.ncl.2014.05.002
4. Nakamura A, Shiba N, Miyazaki D et al (2017) Comparison of the phenotypes of patients harboring in-frame deletions starting at exon 45 in the Duchenne muscular dystrophy gene indicates potential for the development of exon skipping therapy. J Hum Genet 62(4):459–463. https://doi.org/10.1038/jhg.2016.152
5. Nakamura A, Fueki N, Shiba N et al (2016) Deletion of exons 3-9 encompassing a mutational hot spot in the DMD gene presents an asymptomatic phenotype, indicating a target region for multiexon skipping therapy. J Hum Genet 61(7):663–667. https://doi.org/10.1038/jhg.2016.28
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