Presentation Information
[O18-05]Antisense oligonucleotide-mediated skipping of an 84-bp pseudoexon restores PCC activity in propionic acidemia
Eriko Totsune1, Yoichi Wada1,2, Yasuko Mikami-Saito1, Natsuko Arai-Ichinoi1, Naoya Saijo1, Jun Takayama3,4,5,6, Yasuhiro Maeda7, Yoko Nakajima8, Osamu Ohara9, Shigeo Kure1,6,10, Atsuo Kikuchi1,6 (1. Department of Pediatrics, Tohoku University Graduate School of Medicine, 2. Department of Medical Science and Innovation, SiRIUS Institute of Medical Research, Tohoku University, 3. Department of AI and Innovative Medicine, Tohoku University Graduate School of Medicine, 4. Tohoku Medical Megabank organization, Tohoku University, 5. Statistical Genetics Team, RIKEN Center for Advanced Intelligence Project, 6. Department of Rare Disease Genomics, Tohoku University Graduate School of Medicine, 7. Open Facility Center, Fujita Health University, 8. Department of Pediatrics, Fujita Health University School of Medicine, 9. Department of Applied Genomics, Kazusa DNA Research Institute, 10. Miyagi Children's Hospital)
Password required to view
Log in
or