Masaki Mori¹, Takao Morimune^1,2, Yosuke Kadota¹, Faidruz Azura Jam¹, Ayami Tano¹, Yuya Tanaka¹, Haruka Yukiue¹, Sayumi Akabane¹, Mayu Fukumura¹ (1.Molecular Neuroscience Research Center(MNRC), Shiga University of Medical Science, Japan, 2.Department of pediatrics, Shiga University of Medical Science, Japan)

Daisuke Ieda¹, Kohji Kato^1,2, Yuji Nakamura¹, Ikumi Hori¹, Kei Ohashi¹, Yutaka Negishi¹, Ayako Hattori¹, Shinji Saitoh¹ (1.Department of Pediatrics and Neonatology, Nagoya City University Graduate School of Medical Sciences, Nagoya, Japan, 2.Department of Pediatrics, Nagoya University Graduate School of Medicine, Nagoya, Japan)

Mariko Taniguchi-Ikeda^1,2, Makiko Tsutsumi², Yoshinobu Oyazato³, Atsushi Nishiyama³, Shunpei Uchino^4,5, Masakazu Mimaki⁵, Hiroki Kurahashi^1,2 (1.Department of Clinical Genetics, Fujita Health University Hospital, Aichi, Japan, 2.Department of Molecular Genetics, Fujita Health University, Aichi, Japan, 3.Department of Pediatrics, Kakogawa Central City Hospital, Hyogo, Japan, 4.Department of Pediatrics, Graduate School of Medicine, The University of Tokyo, Tokyo, Japan, 5.Department of Pediatrics, Teikyo University Graduate School of Medicine, Tokyo, Japan)

Omar Dabbous, Douglas M. Sproule, Douglas E. Feltner, Marcus Droege, Farid Khan, Ramesh Arjunji (AveXis, Inc., Bannockburn, Illinois, USA)

Yusuke Takezawa¹, Hiromi Fujie², Atsuo Kikuchi¹, Tetsuya Niihori³, Ryo Funayama⁴, Matsuyuki Shirota⁵, Keiko Nakayama⁶, Yoko Aoki³, Masayuki Sasaki⁶, Shigeo Kure¹ (1.Department of Pediatrics, Tohoku University School of Medicine, 2.Department of Pediatrics, Iwaki Kyoritsu General Hospital, Fukushima, Japan, 3.Department of Medical Genetics, Tohoku University School of Medicine, Sendai, Japan, 4.Division of Cell Proliferation, United Centers for Advanced Research and Translational Medicine, Tohoku University Graduate School of Medicine, 5.Division of Interdisciplinary Medical Sciences, United Centers for Advanced Research and Translational Medicine, Tohoku University Graduate School of Medicine, 6.Department of Child Neurology, National Center of Neurology and Psychiatry, Tokyo, Japan)

Hajime Ikehara^1,2, Katsunori Fuji², Toshiyuki Miyashita³, Tomoko Uchida², Tadashi Shiohama², Hideki Uchikawa⁴, Naoki Shimojo², Akihiro Umezawa¹ (1.Center for Regenerative Medicine, National Research Institute for Child health and Development, Tokyo, Japan, 2.Department of Pediatrics, Graduate School of Medicine, Chiba University, Chiba, Japan, 3.Department of Molecular Genetics, Kitasato University School of Medicine, Kanagawa, Japan, 4.Department of Pediatrics, Eastern Chiba Medical Center, Chiba, Japan)