Double Strand Break Free Genome Editing to Target Hematopoietic Stem and Progenitor Cells: Therapeutic Applicability in Fanconi Anemia

被引：0
|
作者：
Ugalde, Laura [1 ]
Karasu, Erman [2 ]
Siegner, Sebastian [2 ]
Garcia, Laura [1 ]
Clemens, Alexandra [2 ]
Olalla, Beatriz [1 ]
Alvarez, Lara [1 ]
Pena, Irene [1 ]
Bueren, Juan A. [1 ]
Corn, Jacob [1 ]
Rio, Paula [1 ]
机构：
[1] CIEMAT CIBERER IIS FJD, Hematopoiet Innovat Therapies Div, Madrid, Spain
[2] Swiss Fed Inst Technol, Mol Hlth Sci, Zurich, Switzerland
来源：
MOLECULAR THERAPY | 2022年 / 30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：
8
引用
收藏
页码：4 / 5
页数：2
相关论文
共 50 条
  • [31] Multipotent hematopoietic cells susceptible to alternative double-strand break repair pathways that promote genome rearrangements
    Francis, Richard
    Richardson, Christine
    GENES & DEVELOPMENT, 2007, 21 (09) : 1064 - 1074
  • [32] Engineering Human Hematopoietic Stem and Progenitor Cells for Lineage-Specific Expression of Galactocerebrosidase Using Genome Editing
    Amorin, Nelson A.
    Golden, Lexi-Ann
    Poletto, Edina
    Johnston, Nicole Marie
    Feist, William
    Menezes, Talia
    Kikuta, Karen
    Luna, Sofia Elena
    Charlton, Celia
    Kashuv, Tyler
    Gomez-Ospina, Natalia
    MOLECULAR THERAPY, 2023, 31 (04) : 782 - 782
  • [33] Efficient GMP-compatible CRISPR-Cas mediated genome editing in hematopoietic stem and progenitor cells
    Turchiano, G.
    Haas, S.
    Hildenbeutel, M.
    Pennucci, V.
    Cornu, T. I.
    Cathomen, T.
    TRANSFUSION MEDICINE AND HEMOTHERAPY, 2017, 44 : 81 - 81
  • [34] LNK (SH2B3) inhibition expands healthy and Fanconi anemia human hematopoietic stem and progenitor cells
    Holdreith, Nicholas
    Lee, Grace
    Chandra, Vemika
    Salinas, Carlo Salas
    Nicholas, Peter
    Olson, Timothy S.
    Tong, Wei
    BLOOD ADVANCES, 2022, 6 (03) : 731 - 745
  • [35] The Sirt1 activator SRT3025 expands hematopoietic stem and progenitor cells and improves hematopoiesis in Fanconi anemia mice
    Zhang, Qing-Shuo
    Deater, Matthew
    Schubert, Kathryn
    Marquez-Loza, Laura
    Pelz, Carl
    Sinclair, David A.
    Grompe, Markus
    STEM CELL RESEARCH, 2015, 15 (01) : 130 - 140
  • [36] Ectopic HOXB4 Overcomes the Inhibitory Effect of TNFα on Hematopoietic Stem and Progenitor Cells in a Murine Fanconi Anemia Model.
    Milsom, Michael D.
    Schiedlmeier, Bernhard
    Bailey, Jeff
    Ali, Abdullah
    Kim, MiOk
    Baum, Christopher
    Jansen, Michael
    Williams, David A.
    BLOOD, 2008, 112 (11) : 711 - 711
  • [37] Correction of SCID-X1 by targeted genome editing of hematopoietic stem/progenitor cells (HSPC) in the mouse model
    Schiroli, G.
    Genovese, P.
    Capo, V.
    Castiello, M. C.
    Albano, L.
    Sanvito, F.
    Holmes, M. C.
    Villa, A.
    Sitia, G.
    Lombardo, A.
    Naldini, L.
    HUMAN GENE THERAPY, 2016, 27 (11) : A135 - A136
  • [38] Transduction of Hematopoietic Stem/Progenitor Cells with a Lentivirus Expressing FANCA off the Human Phosphoglycerate Kinase Promoter Corrects the Fanconi Anemia Phenotype
    Sun, Zejin
    Li, Jingling
    Yang, Yanzhu
    Li, Yan
    Lindemann, Dirk
    Yang, Feng-chun
    Clapp, D. Wade
    Hanenberg, Helmut
    BLOOD, 2011, 118 (21) : 1788 - 1788
  • [39] Correction of SCID-X1 by Targeted Genome Editing of Hematopoietic Stem/Progenitor Cells (HSPC) in the Mouse Model
    Schiroli, Giulia
    Genovese, Pietro
    Capo, Valentina
    Castiello, Maria C.
    Albano, Luisa
    Holmes, Michael C.
    Villa, Anna
    Sitia, Giovanni
    Lombardo, Angelo
    Naldini, Luigi
    MOLECULAR THERAPY, 2016, 24 : S18 - S19
  • [40] Ectopic HOXB4 overcomes the inhibitory effect of tumor necrosis factor-α on Fanconi anemia hematopoietic stem and progenitor cells
    Milsom, Michael D.
    Schiedlmeier, Bernhard
    Bailey, Jeff
    Kim, Mi-Ok
    Li, Dandan
    Jansen, Michael
    Ali, Abdullah Mahmood
    Kirby, Michelle
    Baum, Christopher
    Fairbairn, Leslie J.
    Williams, David A.
    BLOOD, 2009, 113 (21) : 5111 - 5120
12345