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Immortalized Canine Dystrophic Myoblast Cell Lines for Development of Peptide-Conjugated Splice-Switching Oligonucleotides
Duchenne muscular dystrophy (DMD) is a severe muscle-wasting disease caused by frameshift or nonsense mutations in the DMD gene, resulting in the loss of dystrophin from muscle membranes. Exon skipping using splice-switching oligonucleotides (SSOs) restores the reading frame of DMD pre-mRNA by gener...
Autores principales: | Tone, Yuichiro, Mamchaoui, Kamel, Tsoumpra, Maria K., Hashimoto, Yasumasa, Terada, Reiko, Maruyama, Rika, Gait, Michael J., Arzumanov, Andrey A., McClorey, Graham, Imamura, Michihiro, Takeda, Shin'ichi, Yokota, Toshifumi, Wood, Matthew J.A., Mouly, Vincent, Aoki, Yoshitsugu |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Mary Ann Liebert, Inc., publishers
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7997716/ https://www.ncbi.nlm.nih.gov/pubmed/33567244 http://dx.doi.org/10.1089/nat.2020.0907 |
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