^○Tae Matsumoto^1,2, Dongwei Zhao², Noriko Miyake³, Sonoko Narisawa⁴, Jos? Luis Mill?n⁴, Koichi Miyake² (1.Department of Pediatrics, Nippon Medical School, Tokyo, 2.Department of Gene Therapy, Nippon Medical School, Tokyo, 3.Department of Biochemistry and Molecular Biology, Nippon Medical School, Tokyo, 4.Sanford Children’s Health Research Center, Sanford-Burnham-Prebys Medical Discovery Institute, La Jolla, CA.)

^○Karin Kojima, Yoshie Kurokawa, Chika Watanabe, Takanori Yamagata (Department of Pediatrics, Jichi Medical University)

^○Yohta Shimada¹, Takashi Higuchi¹, Saki Matsushima¹, Toya Ohashi², Hiroshi Kobayashi¹ (1.Division of Gene Therapy, Research Center for Medical Sciences, The Jikei University School of Medicine, 2.Department of Human Health Science and Therapeutics, The Jikei University School of Nursing)

^○Toru Uchiyama (Division of Molecular Pathogenesis, Department of Human Genetics, National Center for Child Health and Development)

^○Naoya Uchida (National Institutes of Health)