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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Vydáno v:Proc Natl Acad Sci U S A
Hlavní autoři: 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
Médium: Artigo
Jazyk:Inglês
Vydáno: National Academy of Sciences 2017
Témata:
Biological Sciences
On-line přístup: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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