1. From the Department of Human Gene Therapy and Department of Pediatrics, Hokkaido University School of Medicine, Sapporo, Japan; Department of Pediatrics, Kansai Medical University, Osaka, Japan; Department of Pediatrics, Kawasaki Medical School, Kurashiki, Japan; Hokkaido Red Cross Blood Center, Sapporo, Japan; and Department of Medicine and Biochemistry, Duke University Medical Center, Durham, NC.