Lentiviral Vector-Mediated Stem Cell Gene Therapy for Mucopolysaccharidosis Type IIIA (MPSIIIA)

被引：0
|
作者：
Langford-Smith, A. W. W. [1 ]
Wilkinson, F. L. [1 ]
Langford-Smith, K. J. [1 ]
Bennett, W. [1 ]
Howe, S. [2 ]
Thrasher, A. [2 ]
Hemsley, K. [3 ]
Hopwood, J. [6 ]
Wraith, J. E. [4 ]
Wynn, R. F. [5 ]
Bigger, B. W. [1 ]
机构：
[1] Univ Manchester, MPS Stem Cell Res Grp, Clin Lab Sci, Manchester, Lancs, England
[2] UCL, Ctr Immunodeficiency, Mol Immunol Unit, Inst Child Hlth, London, England
[3] Adelaide Womens & Childrens Hosp, LDRU, Adelaide, SA, Australia
[4] St Marys Hosp, Manchester M13 0JH, Lancs, England
[5] Royal Manchester Childrens Hosp, Blood & Marrow Transplant Unit, Manchester M27 1HA, Lancs, England
[6] Adelaide Womens & Childrens Hosp, DRU, Adelaide, SA, Australia
来源：
HUMAN GENE THERAPY | 2010年 / 21卷 / 04期
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：
引用
收藏
页码：502 / 503
页数：2
相关论文
共 50 条
  • [41] Characterization of lentiviral vector-mediated gene transfer in adult mouse brain
    Baekelandt, V
    Claeys, A
    Eggermont, K
    Lauwers, E
    De Strooper, B
    Nuttin, B
    Debyser, Z
    HUMAN GENE THERAPY, 2002, 13 (07) : 841 - 853
  • [42] Molecular evidence of efficient lentiviral vector-mediated gene transfer into differentiated cardiovascular cells and undifferentiated mesenchymal stem cells for the cardiovascular gene therapy
    Kim, ST
    Kwon, JS
    Li, M
    Kim, YH
    Piao, H
    Youn, TJ
    Kim, DW
    Cho, MC
    EUROPEAN HEART JOURNAL, 2003, 24 : 340 - 340
  • [43] Characterization of a novel mucopolysaccharidosis type II mouse model and recombinant AAV2/8 vector-mediated gene therapy
    Jung, Sung-Chul
    Park, Eun-Sook
    Choi, Eun Nam
    Kim, Chi Hwa
    Kim, Su Jin
    Jin, Dong-Kyu
    MOLECULES AND CELLS, 2010, 30 (01) : 13 - 18
  • [44] Hematopoietic stem cell gene therapy for mucopolysaccharidosis type IIIC
    Badell-Grau, Rafael
    Pakravesh, Kasra
    Sivakumar, Anusha
    Losay, Pauline
    Duong, Kalvin
    Tran, Christine
    Pithia, Rushil
    Khare, Veenita
    Thai, Kevin
    Cherqui, Stephanie
    MOLECULAR GENETICS AND METABOLISM, 2023, 138 (02) : 13 - 13
  • [45] Hematopoietic Stem Cell Gene Therapy For Mucopolysaccharidosis Type IIIC
    Badell-Grau, Rafael A.
    Rainaldi, Joseph
    Losay, Pauline
    Doung, Kalvin
    Pithia, Rushil
    Pakravesh, Kasra
    Silva, Alexander
    Thai, Eric
    Sharma, Jay
    Paulchakrabarti, Mousumi
    Khare, Veenita
    Cherqui, Stephanie
    MOLECULAR THERAPY, 2022, 30 (04) : 300 - 301
  • [46] Hematopoietic Stem Cell Gene Therapy for Mucopolysaccharidosis Type IIIC
    Badell-Grau, Rafael A.
    Sivakumar, Anusha
    Khare, Veenita
    Duong, Kalvin
    Losay, Pauline
    Pakravesh, Kasra
    Tran, Christine
    Pithia, Rushil
    Lengsfield, Mira
    Thai, Kevin
    Sharma, Jay
    Chen, Rola
    Rainaldi, Joseph N.
    Cherqui, Stephanie
    MOLECULAR THERAPY, 2023, 31 (04) : 325 - 326
  • [47] Hematopoietic Stem Cell Gene Therapy for Mucopolysaccharidosis Type IIIC
    Badell-Grau, Rafael A.
    Pakravesh, Kasra
    Chen, Rola
    Thai, Eric
    Son, Frankie
    Duong, Kalvin
    Losay, Pauline
    Pithia, Rushil
    Sivakumar, Anusha
    Khare, Veenita
    Corl, Alexis N.
    Tran, Christine
    Esko, Jefferey
    Cherqui, Stephanie
    MOLECULAR THERAPY, 2024, 32 (04) : 420 - 420
  • [48] Vector-mediated cancer gene therapy - An overview
    Seth, P
    CANCER BIOLOGY & THERAPY, 2005, 4 (05) : 512 - 517
  • [49] Clinical improvements in mucopolysaccharidosis I mice after adult retroviral vector-mediated gene therapy with immunomodulation
    Ponder, Katherine
    Ma, Xiucui
    Liu, Yuli
    Tittiger, Mindy
    Hennig, Anne
    Kovacs, Attila
    Popelka, Sarah
    Herati, Ramin
    Bigg, Mark
    MOLECULAR GENETICS AND METABOLISM, 2007, 92 (04) : S34 - S34
  • [50] Ex vivo lentiviral gene therapy for mucopolysaccharidosis type IVA
    Celik, Betul
    Rintz, Estera
    Nidhi, Fnu
    Khan, Shaukat
    Tomatsu, Shunji
    MOLECULAR GENETICS AND METABOLISM, 2024, 141 (02)
12345