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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Bibliografische gegevens
Gepubliceerd in:Mol Ther
Hoofdauteurs: Lim, Kenji Rowel Q., Echigoya, Yusuke, Nagata, Tetsuya, Kuraoka, Mutsuki, Kobayashi, Masanori, Aoki, Yoshitsugu, Partridge, Terence, Maruyama, Rika, Takeda, Shin’ichi, Yokota, Toshifumi
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: American Society of Gene & Cell Therapy 2019
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Original Article
Online toegang: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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