AOC 1044 induces exon 44 skipping and restores dystrophin protein in preclinical models of Duchenne muscular dystrophy.
AOC 1044 可诱导外显子 44 跳跃，并在杜氏肌营养不良症的临床前模型中恢复肌营养不良蛋白
期刊:Nucleic Acids Research
影响因子:13.1
doi:10.1093/nar/gkaf241
Etxaniz Usue, Marks Isaac, Albin Tyler, Diaz Matthew, Bhardwaj Raghav, Anderson Aaron, Tyaglo Olecya, Hoang Tiffany, Missinato Maria Azzurra, Svensson Kristoffer, Badillo Ben, Kovach Philip R, Leung Laura, Cochran Michael, Kwon Hae Won, Ahad Shah Md Nur, Maruyama Rika, Yokota Toshifumi, Doppalapudi Venkata R, Darimont Beatrice, Younis Husam S, Flanagan W Michael, Levin Arthur A, Huang Hanhua, Karamanlidis Georgios
