Use of Tricyclo-DNA Antisense Oligonucleotides for Exon Skipping

Methods in Molecular Biology
Karima Relizani, Aurélie Goyenvalle

Abstract

Antisense oligonucleotides (AONs) have been actively developed for more than 30 years as a form of molecular medicine and represent promising therapeutic tools for many disorders. Significant progress has been made toward their clinical development in particular for splice switching AONs for the treatment of neuromuscular disorders such as Duchenne muscular dystrophy (DMD). Many different chemistries of AONs can be used for splice switching modulation, and some of them have now reached regulatory approval. However, despite advances in AON chemistry and design, systemic use of AONs is limited due to poor tissue uptake and sufficient therapeutic efficacy is difficult to achieve. Therefore, there is still a critical need to develop efficient AONs able to target all relevant tissues and international efforts are currently on going to advance new compounds or alternative chemistries with higher therapeutic potential. Here we describe the methods to evaluate the potency of tricyclo-DNA (tcDNA)-AONs, a novel class of AONs which displays unique pharmacological properties and unprecedented uptake in many tissues after systemic administration (Goyenvalle et al., Nat Med 21:270-275, 2015; Goyenvalle et al., J Neuromuscul Dis 3:157-167, 20...Continue Reading

Citations

Dec 14, 2018·Journal of Personalized Medicine·Yusuke EchigoyaToshifumi Yokota

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