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
  • [21] Hematopoietic stem cell gene therapy
    Emery, DW
    Nishino, T
    Murata, K
    Fragkos, M
    Stamatoyannopoulos, G
    [J]. INTERNATIONAL JOURNAL OF HEMATOLOGY, 2002, 75 (03) : 228 - 236
  • [22] Hematopoietic stem cell gene therapy
    Negre, O.
    [J]. HUMAN GENE THERAPY, 2018, 29 (12) : A6 - A6
  • [23] Hematopoietic Stem Cell Gene Therapy
    David W. Emery
    Tamon Nishino
    Ken Murata
    Michalis Fragkos
    George Stamatoyannopoulos
    [J]. International Journal of Hematology, 2002, 75 : 228 - 236
  • [24] Long-Term Efficacy and Safety in Preclinical Hematopoietic Stem Cell Gene Therapy in Pompe Mice
    van Til, Niek P.
    Yoon, John K.
    Schindler, Rena
    Dogan, Yildirim
    Barese, Cecilia
    Unnisa, Zeenath
    Jacobs, Mary
    Pizzurro, Daniella
    Oborski, Christine
    Rothe, Michael
    Schambach, Axel
    Yan, Aimin
    Biasco, Luca
    Pfeifer, Richard
    Mason, Chris
    [J]. MOLECULAR THERAPY, 2022, 30 (04) : 492 - 493
  • [25] Towards therapy of chronic granulomatous disease by gene transfer into hematopoietic stem cells
    Ott, MG
    Grez, M
    Becker, S
    Wasser, S
    Hossle, JP
    Seger, RA
    Dinauer, MC
    Hoelzer, D
    [J]. BRITISH JOURNAL OF HAEMATOLOGY, 1998, 102 (01) : 80 - 81
  • [26] Long-term hematopoietic stem cell lentiviral gene therapy rescues neuromuscular manifestations in preclinical study of Pompe disease mice
    van Til, Niek P.
    Schindler, Jeffrey W.
    Yoon, John K.
    Dogan, Yildirim
    Barese, Cecilia
    Unnisa, Zeenath
    Guda, Swaroopa
    Jacobs, Mary E.
    Pizzurro, Daniella
    Trame, Mirjam
    Maiwald, Tim
    Oborski, Christine
    Rothe, Michael
    Schambach, Axel
    Harper, Claudia
    Pfeifer, Richard
    Mason, Chris
    [J]. MOLECULAR GENETICS AND METABOLISM, 2022, 135 (02) : S122 - S123
  • [27] Hematopoietic-Stem-Cell-Based Gene Therapy for HIV Disease
    Kiem, Hans-Peter
    Jerome, Keith R.
    Deeks, Steven G.
    McCune, Joseph M.
    [J]. CELL STEM CELL, 2012, 10 (02) : 137 - 147
  • [28] Hematopoietic stem cell expansion and gene therapy
    Watts, Korashon Lynn
    Adair, Jennifer
    Kiem, Hans-Peter
    [J]. CYTOTHERAPY, 2011, 13 (10) : 1164 - 1171
  • [29] 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
  • [30] Pompe disease gene therapy
    Byrne, Barry J.
    Falk, Darin J.
    Pacak, Christina A.
    Nayak, Sushrusha
    Herzog, Roland W.
    Elder, Melissa E.
    Collins, Shelley W.
    Conlon, Thomas J.
    Clement, Nathalie
    Cleaver, Brian D.
    Cloutier, Denise A.
    Porvasnik, Stacy L.
    Islam, Saleem
    Elmallah, Mai K.
    Martin, Anatole
    Smith, Barbara K.
    Fuller, David D.
    Lawson, Lee Ann
    Mah, Cathryn S.
    [J]. HUMAN MOLECULAR GENETICS, 2011, 20 : R61 - R68