Efficient engraftment of genetically modified cells is necessary to ameliorate central nervous system involvement of murine model of mucopolysaccharidosis type II by hematopoietic stem cell targeted gene therapy.

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第一作者： Saori,Miwa

第一单位： Division of Gene Therapy, Research Center for Medical Sciences, The Jikei University School of Medicine, Tokyo, Japan; Department of Pediatrics, The Jikei University School of Medicine, Tokyo, Japan.

作者： Saori,Miwa ^[1] ; Ayako M,Watabe ^[2] ; Yohta,Shimada ^[3] ; Takashi,Higuchi ^[3] ; Hiroshi,Kobayashi ^[1] ; Takahiro,Fukuda ^[4] ; Fusao,Kato ^[5] ; Hiroyuki,Ida ^[1] ; Toya,Ohashi ^[6]

作者单位： Division of Gene Therapy, Research Center for Medical Sciences, The Jikei University School of Medicine, Tokyo, Japan; Department of Pediatrics, The Jikei University School of Medicine, Tokyo, Japan. ^[1] Institute of Clinical Medicine and Research, Research Center for Medical Sciences, The Jikei University School of Medicine, Tokyo, Japan. ^[2] Division of Gene Therapy, Research Center for Medical Sciences, The Jikei University School of Medicine, Tokyo, Japan. ^[3] Division of Neuropathology, Department of Pathology, The Jikei University School of Medicine, Tokyo, Japan. ^[4] Division of Neuroscience, Research Center for Medical Sciences, The Jikei University School of Medicine, Tokyo, Japan. ^[5] Division of Gene Therapy, Research Center for Medical Sciences, The Jikei University School of Medicine, Tokyo, Japan; Department of Pediatrics, The Jikei University School of Medicine, Tokyo, Japan. Electronic address: tohashi@jikei.ac.jp. ^[6]

关键词 Ex vivo Gene therapy Hunter syndrome Lentivirus vector Mucopolysaccharidosis type II Preconditioning