Application of a Novel Murine Model of Mucopolysaccharidosis Type II for Developing Human Hematopoietic Stem Cell Gene Therapy

被引:0
|
作者
Shimada, Yohta [1 ]
Ishii, Natsumi [1 ]
Higuchi, Takashi [1 ]
Goto, Motohito [2 ]
Ohashi, Toya [3 ]
Kobayashi, Hiroshi [1 ]
机构
[1] Jikei Univ, Sch Med, Res Ctr Med Sci, Div Gene Therapy, Tokyo, Japan
[2] Cent Inst Expt Anim CIEA, Anim Resource Technol Ctr, Kawasaki, Kanagawa, Japan
[3] Jikei Univ, Sch Nursing, Dept Human Hlth Sci & Therapeut, Tokyo, Japan
关键词
D O I
暂无
中图分类号
Q81 [生物工程学(生物技术)]; Q93 [微生物学];
学科分类号
071005 ; 0836 ; 090102 ; 100705 ;
摘要
234
引用
收藏
页码:114 / 114
页数:1
相关论文
共 50 条
  • [21] Neurodevelopmental benefits of early hematopoietic stem cell transplantation in mucopolysaccharidosis type II
    Collyer, John W.
    Rajan, Deepa
    Poe, Michele
    Szabolcs, Paul
    Escolar, Maria L.
    MOLECULAR GENETICS AND METABOLISM, 2025, 144 (02) : 22 - 23
  • [22] Y Gene modified hematopoietic stem cell transplantation for mucopolysaccharidosis type IIIC
    Badell-Grau, Rafael A.
    Sivakumar, Anusha
    Pakravesh, Kasra
    Chen, Rola
    Duong, Kalvin
    Losay, Pauline
    Khare, Veenita
    Thai, Kevin E.
    Pithia, Rushil
    Son, Frankie
    Corl, Alexis N.
    Tran, Christine
    Cherqui, Stephanie
    MOLECULAR GENETICS AND METABOLISM, 2024, 141 (02)
  • [23] Correction of murine hemophilia A by hematopoietic stem cell gene therapy
    Moayeri, M
    Hawley, TS
    Hawley, RG
    MOLECULAR THERAPY, 2005, 12 (06) : 1034 - 1042
  • [24] Prevention of Neurocognitive Deficit by Ex Vivo Lentiviral Transduction of Hematopoietic Stem Cells in a Murine Model of Mucopolysaccharidosis Type II
    Podetz-Pedersen, Kelly M.
    Karlen, Andrea D.
    Buckvold, Megan P.
    Nan, Zenhong
    Slepushkin, Vladimir
    Roy, Andre
    Yakovief, Galina
    Gong, Zi
    Muenzer, Joseph
    Dropulic, Boro
    Low, Walter C.
    McIvor, R. Scott
    MOLECULAR THERAPY, 2013, 21 : S191 - S191
  • [25] Hematopoietic Stem Cell Transplantation for Patients with Mucopolysaccharidosis II
    Kubaski, Francyne
    Yabe, Hiromasa
    Suzuki, Yasuyuki
    Seto, Toshiyuki
    Hamazaki, Takashi
    Mason, Robert W.
    Xie, Li
    Hugo Onsten, Tor Gunnar
    Leistner-Segal, Sandra
    Giugliani, Roberto
    Vu Chi Dung
    Can Thi Bich Ngoc
    Yamaguchi, Seiji
    Montano, Adriana M.
    Orii, Kenji E.
    Fukao, Toshiyuki
    Shintaku, Haruo
    Orii, Tadao
    Tomatsu, Shunji
    BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 2017, 23 (10) : 1795 - 1803
  • [26] Characterization of an immunodeficient mouse model of mucopolysaccharidosis type I suitable for preclinical testing of human stem cell and gene therapy
    Garcia-Rivera, Mayra F.
    Colvin-Wanshura, Leah E.
    Nelson, Matthew S.
    Nan, Zhenhong
    Khan, Shaukat A.
    Rogers, Tyson B.
    Maitra, Indrani
    Low, Walter C.
    Gupta, Pankaj
    BRAIN RESEARCH BULLETIN, 2007, 74 (06) : 429 - 438
  • [27] Lentiviral mediated stem cell gene therapy corrects a mouse model of mucopolysaccharidosis type IIIA
    Bigger, Brian
    Langford-Smith, Alex
    Wilkinson, Fiona
    Langford-Smith, Kia
    Sergijenko, Ana
    Wraith, Ed
    Wynn, Rob
    MOLECULAR GENETICS AND METABOLISM, 2011, 102 (02) : S8 - S8
  • [28] Ex vivo lentiviral gene transfer targeting hematopoietic stem cells for therapy of mucopolysaccharidosis type II, Hunter syndrome
    McIvor, R. Scott
    Wolf, Daniel A.
    Podetz-Petersen, Kelly
    Nan, Zhenhong
    Whitley, Chester B.
    Muenzer, Joseph
    Orchard, Paul J.
    Low, Walter C.
    MOLECULAR GENETICS AND METABOLISM, 2009, 96 (02) : S30 - S31
  • [29] Brain targeted stem cell gene therapy corrects mucopolysaccharidosis type II via multiple mechanisms
    Gleitz, H.
    Liao, A.
    Cook, J.
    Rowlston, S.
    Forte, G.
    D'Souza, Z.
    O'Leary, C.
    Holley, R.
    Bigger, B.
    HUMAN GENE THERAPY, 2018, 29 (12) : A28 - A28
  • [30] Ex vivo gene therapy improves the accumulation of glycosaminoglycans in brain from murine model of mucopolysaccharidosis type II
    Wakabayashi, Taichi
    Shimada, Yohta
    Higuchi, Takashi
    Kobayashi, Hiroshi
    Ida, Hiroyuki
    Ohashi, Toya
    GLYCOBIOLOGY, 2014, 24 (11) : 1214 - 1214