Art

J-GLOBAL ID：202002224524399351   Reference number：20A1523748

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

遺伝子組み換え細胞の効率的な移植は造血幹細胞標的遺伝子治療によるムコ多糖症II型のマウスモデルの中枢神経系関与を改善するために必要である【JST・京大機械翻訳】

Author (13)： Miwa Saori (Division of Gene Therapy, Research Center for Medical Sciences, The Jikei University School of Medicine, Tokyo, Japan) ,  Miwa Saori (Department of Pediatrics, The Jikei University School of Medicine, Tokyo, Japan) ,  Watabe Ayako M. (Institute of Clinical Medicine and Research, Research Center for Medical Sciences, The Jikei University School of Medicine, Tokyo, Japan) ,  Shimada Yohta (Division of Gene Therapy, Research Center for Medical Sciences, The Jikei University School of Medicine, Tokyo, Japan) ,  Higuchi Takashi (Division of Gene Therapy, Research Center for Medical Sciences, The Jikei University School of Medicine, Tokyo, Japan) ,  Kobayashi Hiroshi (Division of Gene Therapy, Research Center for Medical Sciences, The Jikei University School of Medicine, Tokyo, Japan) ,  Kobayashi Hiroshi (Department of Pediatrics, The Jikei University School of Medicine, Tokyo, Japan) ,  Fukuda Takahiro (Division of Neuropathology, Department of Pathology, The Jikei University School of Medicine, Tokyo, Japan) ,  Kato Fusao (Division of Neuroscience, Research Center for Medical Sciences, The Jikei University School of Medicine, Tokyo, Japan) ,  Ida Hiroyuki (Division of Gene Therapy, Research Center for Medical Sciences, The Jikei University School of Medicine, Tokyo, Japan) ,  Ida Hiroyuki (Department of Pediatrics, The Jikei University School of Medicine, Tokyo, Japan) ,  Ohashi Toya (Division of Gene Therapy, Research Center for Medical Sciences, The Jikei University School of Medicine, Tokyo, Japan) ,  Ohashi Toya (Department of Pediatrics, The Jikei University School of Medicine, Tokyo, Japan)
Material： Molecular Genetics and Metabolism  (Mol Genet Metab)
Volume： 130  Issue： 4  Page： 262-273  Publication year： 2020 
JST Material Number： E0820A  ISSN： 1096-7192  Document type： Article
Article type： 原著論文  Country of issue： Netherlands (NLD)  Language： ENGLISH (EN)

Abstract/Point： Mucopolysaccharidosis type II ...

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Thesaurus term： enzyme ,  *heredity ,  *gene ,  enzyme activity ,  mortality ,  sulfuric ester hydrolase ,  *central nervous system ,  lethal irradiation ,  lysosome ,  *hematopoietic stem cell ,  gene therapy ,  monoclonal antibody ,  targeting ,  hematopoietic stem cell transplantation
Semi thesaurus term： *マウスモデル , 【Automatic Indexing@JST】

Author keywords (6)： Mucopolysaccharidosis type II ,  Hunter syndrome ,  Gene therapy ,  Preconditioning ,  Lentivirus vector ,  Ex vivo

JST classification (2)： Treatment for congenital diseases,deformities.  (GD03030R) ,  Treatment for metabolic diseases,nutritional diseases.  (GD05030F)

Terms in the title (9)： 遺伝子 ,  組み換え細胞 ,  移植 ,  造血幹細胞 ,  標的遺伝子 ,  治療 ,  ムコ多糖症II型 ,  マウスモデル ,  中枢神経系