Hematopoietic stem cell gene therapy using a selective amplifier gene in a murine X-CGD model.

被引:0
|
作者
Hara, T
Kume, A
Hanazono, Y
Mizukami, HI
Okada, T
Tsurumi, H
Moriwaki, H
Hasegawa, M
Ozawa, K
机构
[1] Jichi Med Sch, Div Genet Therapeut, Kawati, Tochigi, Japan
[2] Gifu Univ, Sch Med, Dept Internal Med 1, Gifu 500, Japan
[3] DNAVEC Res Inc, Tsukuba, Ibaraki, Japan
关键词
D O I
暂无
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
432
引用
收藏
页码:117A / 117A
页数:1
相关论文
共 50 条
  • [41] Lentiviral Hematopoietic Stem Cell Gene Therapy Corrects Murine Pompe Disease
    Stok, Merel
    de Boer, Helen
    Huston, Marshall W.
    Jacobs, Edwin H.
    Roovers, Onno
    Visser, Trudi P.
    Jahr, Holger
    Duncker, Dirk J.
    van Deel, Elza D.
    Reuser, Arnold J. J.
    van Til, Niek P.
    Wagemaker, Gerard
    [J]. MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2020, 17 : 1014 - 1025
  • [42] Alpharetroviral Vector-mediated Gene Therapy for X-CGD: Functional Correction and Lack of Aberrant Splicing
    Kaufmann, Kerstin B.
    Brendel, Christian
    Suerth, Julia D.
    Mueller-Kuller, Uta
    Chen-Wichmann, Linping
    Schwaeble, Joachim
    Pahujani, Shweta
    Kunkel, Rana
    Schambach, Axel
    Baum, Christopher
    Grez, Manuel
    [J]. MOLECULAR THERAPY, 2013, 21 (03) : 648 - 661
  • [43] Lentiviral CYBB Gene Therapy Restores Oxidase Activity and Relieves Disease Symptoms in an X-CGD Infant
    Chang, Lung-Ji
    Sun, Yuan
    Tsao, Shih-Ting
    Xiao, Juan
    Zhang, Rui
    Gui, Siqian
    Zheng, Jie
    He, Jian-Xing
    Zheng, Huyong
    [J]. MOLECULAR THERAPY, 2019, 27 (04) : 393 - 393
  • [44] Lentiviral Hematopoietic Stem Cell Gene Therapy Rescues Clinical Phenotypes in a Murine Model of Pompe Disease
    Piras, Giuseppa
    Montiel-Equihua, Claudia
    Chan, Yee-Ka Agnes
    Wantuch, Slawomir
    Stuckey, Daniel
    Burke, Derek
    Prunty, Helen
    Phadke, Rahul
    Chambers, Darren
    Partida-Gaytan, Armando
    Leon-Rico, Diego
    Panchal, Neelam
    Whitmore, Kathryn
    Calero, Miguel
    Benedetti, Sara
    Santilli, Giorgia
    Thrasher, Adrian J.
    Gaspar, H. Bobby
    [J]. MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2020, 18 : 558 - 570
  • [45] Hematopoietic Stem Cell Gene Therapy Corrects Neuropathic Phenotype in Murine Model of Mucopolysaccharidosis Type II
    Wakabayashi, Taichi
    Shimada, Yohta
    Akiyama, Kazumasa
    Higuchi, Takashi
    Fukuda, Takahiro
    Kobayashi, Hiroshi
    Eto, Yoshikatsu
    Ida, Hiroyuki
    Ohashi, Toya
    [J]. HUMAN GENE THERAPY, 2015, 26 (06) : 357 - 366
  • [46] Curative in vivo hematopoietic stem cell gene therapy of murine thalassemia using large regulatory elements
    Wang, Hongjie
    Georgakopoulou, Aphrodite
    Li, Chang
    Liu, Zhinan
    Gil, Sucheol
    Bashyam, Ashvin
    Yannaki, Evangelia
    Anagnostopoulos, Achilles
    Pande, Amit
    Izsvak, Zsuzsanna
    Papayannopoulou, Thalia
    Lieber, Andre
    [J]. JCI INSIGHT, 2020, 5 (16)
  • [47] Vector silencing in a gene therapy trial for X-CGD and strategies to improve long-term transgene expression
    Schultze-Strasser, Stephan
    Brendel, Christian
    Kramer, Rolf
    Preiss, Carolin
    Kunkel, Hana
    Stein, Stefan
    Grez, Manuel
    [J]. HUMAN GENE THERAPY, 2009, 20 (11) : 1439 - 1439
  • [48] Hematopoietic Stem Cell Gene Therapy for Cystinosis
    Ur, Sarah
    Rocca, Celine J.
    Sharma, Jay
    Kohn, Donald B.
    Cherqui, Stephanie
    [J]. MOLECULAR THERAPY, 2013, 21 : S190 - S191
  • [49] Hematopoietic stem cell expansion and gene therapy
    Watts, Korashon Lynn
    Adair, Jennifer
    Kiem, Hans-Peter
    [J]. CYTOTHERAPY, 2011, 13 (10) : 1164 - 1171
  • [50] Gene therapy using hematopoietic stem cells
    Kohn, DB
    [J]. CURRENT OPINION IN MOLECULAR THERAPEUTICS, 1999, 1 (04) : 437 - 442