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

Goyenvalle A, Griffith G, Babbs A, El Andaloussi S, Ezzat K, Avril A, et al. Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers. Nature medicine. 2015;21(3):270-5   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 […]