Towards hematopoietic stem cell gene therapy for the treatment of Pompe disease

被引:0
|
作者
Stok, M. [1 ]
de Boer, H. [1 ]
de Visser, T. P. [1 ]
Reuser, A. J. J. [1 ]
van Deel, E. [1 ]
Duncker, D. J. [1 ]
van Til, N. P. [1 ]
Wagemaker, G. [1 ]
机构
[1] Erasmus MC, Rotterdam, Netherlands
关键词
D O I
10.1016/j.nmd.2011.06.1002
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
引用
收藏
页码:715 / 715
页数:1
相关论文
共 50 条
  • [31] Partial phenotypic correction and immune tolerance induction to enzyme replacement therapy after hematopoietic stem cell gene transfer of α-glucosidase in Pompe disease
    Douillard-Guilloux, Gaelle
    Richard, Emmanuel
    Batista, Lionel
    Caillaud, Catherine
    [J]. JOURNAL OF GENE MEDICINE, 2009, 11 (04): : 279 - 287
  • [32] Towards a Mechanistic Understanding of Ex Vivo Hematopoietic Stem Cell Expansion for Gene Therapy
    Cammarata, Andrea
    Desantis, Giacomo
    Zonari, Erika
    Cittaro, Davide
    Naldini, Matteo Maria
    Gentner, Bernhard
    [J]. MOLECULAR THERAPY, 2018, 26 (05) : 18 - 19
  • [33] Hematopoietic Stem Cell Gene-Addition/Editing Therapy in Sickle Cell Disease
    Germino-Watnick, Paula
    Hinds, Malikiya
    Le, Anh
    Chu, Rebecca
    Liu, Xiong
    Uchida, Naoya
    [J]. CELLS, 2022, 11 (11)
  • [34] Hematopoietic Stem Cell Gene Therapy for Storage Disease: Current and New Indications
    Biffi, Alessandra
    [J]. MOLECULAR THERAPY, 2017, 25 (05) : 1155 - 1162
  • [35] Coreceptor-Based Hematopoietic Stem Cell Gene Therapy for HIV Disease
    Hu, JinTing
    Feng, YeWen
    Ma, Ping
    Lai, Yu
    [J]. CURRENT STEM CELL RESEARCH & THERAPY, 2019, 14 (07) : 591 - 597
  • [36] Long-term hematopoietic stem cell gene therapy corrects neuromuscular manifestations in preclinical study of Pompe mice
    van Til, Niek P.
    Dogan, Yildirim
    Barese, Cecilia
    Unnisa, Zeenath
    Guda, Swaroopa
    Schindler, Rena
    Yoon, John
    Jacobs, Mary
    Chiyyeadu, Abhishek
    Pizzurro, Daniella
    Liu, Bianling
    Braun, Maurine
    Oborski, Christine
    Schambach, Axel
    Harper, Claudia
    Pfeifer, Richard
    Mason, Chris
    [J]. MOLECULAR GENETICS AND METABOLISM, 2021, 132 (02) : S107 - S107
  • [37] Assessment of Gene Therapy Treatment in the Pompe Disease Canine Model
    Pope, Megan K.
    Coleman, Kirsten
    Wichman, Megan
    Cloutier, Denise
    Cousins, Christina
    Mueller, Lee Ann
    Whitehead, Victoria
    Meena, Naresh
    Raben, Nina
    Corti, Manuela
    Byrne, Barry
    [J]. MOLECULAR THERAPY, 2022, 30 (04) : 2 - 3
  • [38] Hematopoietic stem cell mobilization strategies for gene therapy of beta thalassemia and sickle cell disease
    Yannaki, Evangelia
    Stamatoyannopoulos, George
    [J]. COOLEY'S ANEMIA: NINTH SYMPOSIUM, 2010, 1202 : 59 - 63
  • [39] Hematopoietic stem cell gene therapy to halt neurodegeneration
    Biffi, Alessandra
    [J]. NEUROTHERAPEUTICS, 2024, 21 (04)
  • [40] Genotoxicity of retroviral hematopoietic stem cell gene therapy
    Trobridge, Grant D.
    [J]. EXPERT OPINION ON BIOLOGICAL THERAPY, 2011, 11 (05) : 581 - 593