Generation of a human p47phox-deficient chronic granulomatous disease cell line using CRISPR/Cas9 for fast gene therapy vector testing

被引:0
|
作者
Wrona, D. [1 ,2 ,3 ]
Siler, U. [1 ,2 ,3 ]
Reichenbach, J. [1 ,2 ,3 ]
机构
[1] Univ Childrens Hosp Zurich, Div Immunol, Zurich, Switzerland
[2] Childrens Res Ctr, Zurich, Switzerland
[3] Univ Zurich, Zurich, Switzerland
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
SSAIP 28
引用
收藏
页码:59S / 59S
页数:1
相关论文
共 50 条
  • [1] CRISPR/Cas9-generated p47phox-deficient cell line for Chronic Granulomatous Disease gene therapy vector development
    Wrona, Dominik
    Siler, Ulrich
    Reichenbach, Janine
    SCIENTIFIC REPORTS, 2017, 7
  • [2] CRISPR/Cas9-generated p47phox-deficient cell line for Chronic Granulomatous Disease gene therapy vector development
    Dominik Wrona
    Ulrich Siler
    Janine Reichenbach
    Scientific Reports, 7
  • [3] Lentiviral SIN-vector for p47phox-deficient Chronic Granulomatous Disease Gene Therapy
    Valencia, R.
    Wrona, D.
    Brendel, C.
    Haenseler, W.
    Santilli, G.
    Thrasher, A.
    Grez, M.
    Reichenbach, J.
    Siler, U.
    HUMAN GENE THERAPY, 2015, 26 (10) : A61 - A62
  • [4] Preclinical Optimization and Safety Studies of a New Lentiviral Gene Therapy for p47phox-Deficient Chronic Granulomatous Disease
    Schejtman, Andrea
    Vetharoy, Winston
    Choi, Uimook
    Rivat, Christine
    Theobald, Narda
    Piras, Giuseppa
    Leon-Rico, Diego
    Buckland, Karen
    Armenteros-Monterroso, Elena
    Benedetti, Sara
    Ashworth, Michael T.
    Rothe, Michael
    Schambach, Axel
    Gaspar, Hubert Bobby
    Kang, Elizabeth M.
    Malech, Harry L.
    Thrasher, Adrian J.
    Santilli, Giorgia
    HUMAN GENE THERAPY, 2021, 32 (17-18) : 949 - 958
  • [5] Gene-edited pseudogene resurrection corrects p47phox-deficient chronic granulomatous disease
    Merling, Randall K.
    Kuhns, Douglas B.
    Sweeney, Colin L.
    Wu, Xiaolin
    Burkett, Sandra
    Chu, Jessica
    Lee, Janet
    Koontz, Sherry
    Di Pasquale, Giovanni
    Afione, Sandra A.
    Chiorini, John A.
    Kang, Elizabeth M.
    Choi, Uimook
    De Ravin, Suk See
    Malech, Harry L.
    BLOOD ADVANCES, 2017, 1 (04) : 270 - 278
  • [6] Applying Cas9/sgRNA-Mediated Targeted Genome Engineering to Generate a P47phox-Deficient Induced Pluripotent Stem Cell Line for Gene Therapy Development
    Klatt, Denise
    Selich, Anton
    Schott, Juliane W.
    Hoffmann, Dirk
    Schambach, Axel
    MOLECULAR THERAPY, 2018, 26 (05) : 378 - 378
  • [7] Lentiviral Gene Therapy for p47phox Deficient Chronic Granulomatous Disease
    Shejtman, Andrea
    Aragao Filho, Walmir Cutrim
    Weisser, Maren
    Zinicola, Marta
    Booth, Claire
    Burns, Siobhan O.
    Siler, Ulrich
    Reichenbach, Janine
    Gaspar, H. Bobby
    Neto, Antonio Condino
    Thrasher, Adrian J.
    Santilli, Giorgia
    MOLECULAR THERAPY, 2017, 25 (05) : 88 - 89
  • [8] Novel Diagnostic Tool for p47phox-Deficient Chronic Granulomatous Disease Patient and Carrier Detection
    Wrona, Dominik
    Siler, Ulrich
    Reichenbach, Janine
    MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2019, 13 : 274 - 278
  • [9] Preclinical evaluation of the pCCLCHIM-p47 lentiviral vector for the gene therapy of p47phox deficient Chronic Granulomatous Disease
    Schejtman, A.
    Vetharoy, W.
    Choi, U.
    Rothe, M.
    Rivat, C.
    Piras, G.
    Benedetti, S.
    Santos, C. C.
    Schambach, A.
    Malech, H. L.
    Thrasher, A. J.
    Santilli, G.
    HUMAN GENE THERAPY, 2019, 30 (11) : A80 - A80
  • [10] Correction of p47-phox deficient Chronic Granulomatous Disease by lentiviral gene therapy
    Schejtman, Andrea
    Cutrim Aragao Filho, W.
    Clare, S.
    Weisser, M.
    Zinicola, M.
    Booth, C.
    Burns, S. O.
    Siler, U.
    Reichenbach, J.
    Gaspar, H. B.
    Thomas, D.
    Condino-Neto, A.
    Thrasher, A. J.
    Santilli, G.
    HUMAN GENE THERAPY, 2018, 29 (07) : A12 - A12