Sensitive system to test antisense oligos for splice therapy in mouse liver

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Jon Moulton
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Sensitive system to test antisense oligos for splice therapy in mouse liver

Gallego-Villar L, Viecelli HM, Pérez B, Harding CO, Ugarte M, Thöny B, Desviat LR. A sensitive assay system to test antisense oligonucleotides for splice suppression therapy in the mouse liver. Mol Ther Nucleic Acids. 2014 Sep 16;3:e193. doi: 10.1038/mtna.2014.44.