Effects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is a lethal genetic disorder caused by an absence of the dystrophin protein in bodywide muscles, including the heart. Cardiomyopathy is a leading cause of death in DMD. Exon skipping via synthetic phosphorodiamidate morpholino oligomers (PMOs) represents one of the...

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Udgivet i:Proc Natl Acad Sci U S A
Main Authors: Echigoya, Yusuke, Nakamura, Akinori, Nagata, Tetsuya, Urasawa, Nobuyuki, Lim, Kenji Rowel Q., Trieu, Nhu, Panesar, Dharminder, Kuraoka, Mutsuki, Moulton, Hong M., Saito, Takashi, Aoki, Yoshitsugu, Iversen, Patrick, Sazani, Peter, Kole, Ryszard, Maruyama, Rika, Partridge, Terry, Takeda, Shin’ichi, Yokota, Toshifumi
Format: Artigo
Sprog:Inglês
Udgivet: National Academy of Sciences 2017
Fag:
Biological Sciences
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5402437/
https://ncbi.nlm.nih.gov/pubmed/28373570
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1613203114
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