Effects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog mod...
Effects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy
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Author / Creator
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 and Yokota, Toshifumi
Publisher
United States: National Academy of Sciences
Journal title
Language
English
Formats
Publication information
Publisher
United States: National Academy of Sciences
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Scope and Contents
Contents
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 most promising therapeutic options, yet PMOs have...
Alternative Titles
Full title
Effects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy
Authors, Artists and Contributors
Author / Creator
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Primary Identifiers
Record Identifier
TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_5402437
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_5402437
Other Identifiers
ISSN
0027-8424
E-ISSN
1091-6490
DOI
10.1073/pnas.1613203114
