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

• Main Authors: Yusuke Echigoya (Author), Yoshitsugu Aoki (Author), Bailey Miskew (Author), Dharminder Panesar (Author), Aleksander Touznik (Author), Tetsuya Nagata (Author), Jun Tanihata (Author), Akinori Nakamura (Author), Kanneboyina Nagaraju (Author), Toshifumi Yokota (Author)
• Format: Book
• Published: Elsevier, 2015-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...