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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Αποθηκεύτηκε σε:
Λεπτομέρειες βιβλιογραφικής εγγραφής
Τόπος έκδοσης:Proc Natl Acad Sci U S A
Κύριοι συγγραφείς: 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
Μορφή: Artigo
Γλώσσα:Inglês
Έκδοση: National Academy of Sciences 2017
Θέματα:
Biological Sciences
Διαθέσιμο Online: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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