Exons 45–55 Skipping Using Mutation-Tailored Cocktails of Antisense Morpholinos in the DMD Gene

Mutations in the dystrophin (DMD) gene and consequent loss of dystrophin cause Duchenne muscular dystrophy (DMD). A promising therapy for DMD, single-exon skipping using antisense phosphorodiamidate morpholino oligomers (PMOs), currently confronts major issues in that an antisense drug induces the p...

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Bibliografiske detaljer
Udgivet i:Mol Ther
Main Authors: Echigoya, Yusuke, Lim, Kenji Rowel Q., Melo, Dyanna, Bao, Bo, Trieu, Nhu, Mizobe, Yoshitaka, Maruyama, Rika, Mamchaoui, Kamel, Tanihata, Jun, Aoki, Yoshitsugu, Takeda, Shin’ichi, Mouly, Vincent, Duddy, William, Yokota, Toshifumi
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2019
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Original Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6838919/
https://ncbi.nlm.nih.gov/pubmed/31416775
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2019.07.012
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