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

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Published in Proc Natl Acad Sci U S A on April 03, 2017

Authors

Yusuke Echigoya1, Akinori Nakamura2,3, Tetsuya Nagata2, Nobuyuki Urasawa2,4, Kenji Rowel Q Lim1, Nhu Trieu1, Dharminder Panesar1, Mutsuki Kuraoka2, Hong M Moulton5, Takashi Saito2, Yoshitsugu Aoki2, Patrick Iversen6, Peter Sazani6, Ryszard Kole6, Rika Maruyama1, Terry Partridge7,8, Shin'ichi Takeda9, Toshifumi Yokota10,11

Author Affiliations

1: Department of Medical Genetics, School of Human Development, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB, Canada T6G 2H7.
2: Department of Molecular Therapy, National Center of Neurology and Psychiatry, National Institute of Neuroscience, Kodaira, Tokyo 187-8502, Japan.
3: Third Department of Internal Medicine, Shinshu University School of Medicine, Matsumoto, Nagano 390-8621, Japan.
4: Department of Cardiovascular Internal Medicine, Nagano Red Cross Hospital, Nagano 380-8582, Japan.
5: Department of Biomedical Sciences, College of Veterinary Medicine, Oregon State University, Corvallis, OR 97331.
6: Sarepta Therapeutics, Inc., Cambridge, MA 02142.
7: Research Center for Genetic Medicine, Children's National Medical Center, Washington, DC 20010.
8: Department of Integrative Systems Biology, George Washington University School of Medicine, Washington, DC 20010.
9: Department of Molecular Therapy, National Center of Neurology and Psychiatry, National Institute of Neuroscience, Kodaira, Tokyo 187-8502, Japan; takeda@ncnp.go.jp toshifum@ualberta.ca.
10: Department of Medical Genetics, School of Human Development, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB, Canada T6G 2H7; takeda@ncnp.go.jp toshifum@ualberta.ca.
11: The Friends of Garrett Cumming Research & Muscular Dystrophy Canada HM Toupin Neurological Science Research Chair, Edmonton, AB, Canada T6G 2H7.

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