Long-Term Efficacy of Systemic Multiexon Skipping Targeting Dystrophin Exons 45–55 With a Cocktail of Vivo-Morpholinos in Mdx52 Mice

Long-Term Efficacy of Systemic Multiexon Skipping Targeting Dystrophin Exons 45–55 With a Cocktail of Vivo-Morpholinos in Mdx52 Mice

Antisense-mediated exon skipping, which can restore the reading frame, is a most promising therapeutic approach for Duchenne muscular dystrophy. Remaining challenges include the limited applicability to patients and unclear function of truncated dystrophin proteins. Multiexon skipping targeting exon...

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Bibliographic Details
Main Authors: Yusuke Echigoya, Yoshitsugu Aoki, Bailey Miskew, Dharminder Panesar, Aleksander Touznik, Tetsuya Nagata, Jun Tanihata, Akinori Nakamura, Kanneboyina Nagaraju, Toshifumi Yokota
Format: Article
Language:English
Published: Elsevier 2015-01-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
antisense therapeutics
Duchenne muscular dystrophy
dystrophin
mdx52 mouse
multiexon skipping
Vivo-Morpholinos
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253116300129

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http://www.sciencedirect.com/science/article/pii/S2162253116300129

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