Efficacy of Multi-exon Skipping Treatment in Duchenne Muscular Dystrophy Dog Model Neonates

Duchenne muscular dystrophy (DMD) is caused by mutations in DMD, which codes for dystrophin. Because the progressive and irreversible degeneration of muscle occurs from childhood, earlier therapy is required to prevent dystrophic progression. Exon skipping by antisense oligonucleotides called phosph...

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Publicat a:Mol Ther
Autors principals: Lim, Kenji Rowel Q., Echigoya, Yusuke, Nagata, Tetsuya, Kuraoka, Mutsuki, Kobayashi, Masanori, Aoki, Yoshitsugu, Partridge, Terence, Maruyama, Rika, Takeda, Shin’ichi, Yokota, Toshifumi
Format: Artigo
Idioma:Inglês
Publicat: American Society of Gene & Cell Therapy 2019
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Original Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC6318815/
https://ncbi.nlm.nih.gov/pubmed/30448197
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2018.10.011
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