Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers View Full Text


Ontology type: schema:ScholarlyArticle     


Article Info

DATE

2015-03

AUTHORS

Aurélie Goyenvalle, Graziella Griffith, Arran Babbs, Samir El Andaloussi, Kariem Ezzat, Aurélie Avril, Branislav Dugovic, Rémi Chaussenot, Arnaud Ferry, Thomas Voit, Helge Amthor, Claudia Bühr, Stefan Schürch, Matthew J A Wood, Kay E Davies, Cyrille Vaillend, Christian Leumann, Luis Garcia

ABSTRACT

Antisense oligonucleotides (AONs) hold promise for therapeutic correction of many genetic diseases via exon skipping, and the first AON-based drugs have entered clinical trials for neuromuscular disorders. However, despite advances in AON chemistry and design, systemic use of AONs is limited because of poor tissue uptake, and recent clinical reports confirm that sufficient therapeutic efficacy has not yet been achieved. Here we present a new class of AONs made of tricyclo-DNA (tcDNA), which displays unique pharmacological properties and unprecedented uptake by many tissues after systemic administration. We demonstrate these properties in two mouse models of Duchenne muscular dystrophy (DMD), a neurogenetic disease typically caused by frame-shifting deletions or nonsense mutations in the gene encoding dystrophin and characterized by progressive muscle weakness, cardiomyopathy, respiratory failure and neurocognitive impairment. Although current naked AONs do not enter the heart or cross the blood-brain barrier to any substantial extent, we show that systemic delivery of tcDNA-AONs promotes a high degree of rescue of dystrophin expression in skeletal muscles, the heart and, to a lesser extent, the brain. Our results demonstrate for the first time a physiological improvement of cardio-respiratory functions and a correction of behavioral features in DMD model mice. This makes tcDNA-AON chemistry particularly attractive as a potential future therapy for patients with DMD and other neuromuscular disorders or with other diseases that are eligible for exon-skipping approaches requiring whole-body treatment. More... »

PAGES

270-275

Identifiers

URI

http://scigraph.springernature.com/pub.10.1038/nm.3765

DOI

http://dx.doi.org/10.1038/nm.3765

DIMENSIONS

https://app.dimensions.ai/details/publication/pub.1014266352

PUBMED

https://www.ncbi.nlm.nih.gov/pubmed/25642938


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