| Chapter title |
Systemic Delivery of Morpholinos to Skip Multiple Exons in a Dog Model of Duchenne Muscular Dystrophy
|
|---|---|
| Chapter number | 17 |
| Book title |
Morpholino Oligomers
|
| Published in |
Methods in molecular biology, April 2017
|
| DOI | 10.1007/978-1-4939-6817-6_17 |
| Pubmed ID | |
| Book ISBNs |
978-1-4939-6815-2, 978-1-4939-6817-6
|
| Authors |
Maruyama, Rika, Echigoya, Yusuke, Caluseriu, Oana, Aoki, Yoshitsugu, Takeda, Shin’ichi, Yokota, Toshifumi, Rika Maruyama, Yusuke Echigoya, Oana Caluseriu, Yoshitsugu Aoki, Shin’ichi Takeda, Toshifumi Yokota |
| Editors |
Hong M. Moulton, Jon D. Moulton |
| Abstract |
Exon-skipping therapy is an emerging approach that uses synthetic DNA-like molecules called antisense oligonucleotides (AONs) to splice out frame-disrupting parts of mRNA, restore the reading frame, and produce truncated yet functional proteins. Multiple exon skipping utilizing a cocktail of AONs can theoretically treat 80-90% of patients with Duchenne muscular dystrophy (DMD). The success of multiple exon skipping by the systemic delivery of a cocktail of AONs called phosphorodiamidate morpholino oligomers (PMOs) in a DMD dog model has made a significant impact on the development of therapeutics for DMD, leading to clinical trials of PMO-based drugs. Here, we describe the systemic delivery of a cocktail of PMOs to skip multiple exons in dystrophic dogs and the evaluation of the efficacies and toxicity in vivo. |
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