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.
CITATION STYLE
Maruyama, R., Echigoya, Y., Caluseriu, O., Aoki, Y., Takeda, S., & Yokota, T. (2017). Systemic delivery of morpholinos to skip multiple exons in a dog model of duchenne muscular dystrophy. In Methods in Molecular Biology (Vol. 1565, pp. 201–213). Humana Press Inc. https://doi.org/10.1007/978-1-4939-6817-6_17
Mendeley helps you to discover research relevant for your work.