AOC 1044 induces exon 44 skipping and restores dystrophin protein in preclinical models of Duchenne muscular dystrophy

Usue Etxaniz, Isaac Marks, Tyler Albin, Matthew Diaz, Raghav Bhardwaj, Aaron Anderson, Olecya Tyaglo, Tiffany Hoang, Maria Azzurra Missinato, Kristoffer Svensson, Ben Badillo, Philip R Kovach, Laura Leung, Michael Cochran, Hae Won Kwon, Md Nur Ahad Shah, Rika Maruyama, Toshifumi Yokota, Venkata R Doppalapudi, Beatrice Darimont, Husam S Younis, W Michael Flanagan, Arthur A Levin, Hanhua Huang, Georgios Karamanlidis
Nucleic Acids Research, Volume 53, Issue 6, 11 April 2025, gkaf241, https://doi.org/10.1093/nar/gkaf241