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

@article{Goyenvalle2015FunctionalCI,
  title={Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers},
  author={Aur{\'e}lie Goyenvalle and Graziella Griffith and Arran Babbs and Samir A El Andaloussi and Kariem Ezzat and Aur{\'e}lie Avril and Branislav Dugovi{\vc} and R{\'e}mi Chaussenot and Arnaud Ferry and Thomas Voit and Helge Amthor and Claudia B{\"u}hr and Stefan Sch{\"u}rch and Matthew J. Wood and K. E. Davies and Cyrille Vaillend and Christian J. Leumann and Luis Garc{\'i}a},
  journal={Nature Medicine},
  year={2015},
  volume={21},
  pages={270-275}
}
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… CONTINUE READING
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