Gene Therapy Approach for Severe Congenital Neutropenia by CRISPR/Cas9 Gene-Editing of Hematopoietic Stem Cells and iPSCs

被引：0
|
作者：
Skokowa, Julia [1 ]
Ritter, Malte [1 ]
Nasri, Masoud [1 ]
Mir, Perihan [1 ]
Dannenmann, Benjamin [1 ]
Klimiankou, Maksim [1 ]
Aghaallaei, Nargessadat [1 ]
Secker, Benjamin [1 ]
Haaf, Jeremy [1 ]
Bravo, Natalia Alejandra Borbaran [1 ]
Zeidler, Cornelia [2 ]
Bajoghli, Baubak [1 ]
Xu, Yun [1 ]
Welte, Karl [1 ]
机构：
[1] Univ Hosp Tuebingen, Clin Immunol, Hematol, Oncol, Tubingen, Germany
[2] MHH, Clin Immunol, Hematol, Oncol, Hannover, Germany
来源：
MOLECULAR THERAPY | 2020年 / 28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：
371
引用
收藏
页码：166 / 167
页数：2
相关论文
共 50 条
  • [41] Gene-ectomy: Gene Ablation with CRISPR/Cas9 in Human Hematopoietic Cells
    Calero-Garcia, Miguel
    Gaspar, H. Bobby
    CELL STEM CELL, 2014, 15 (05) : 529 - 530
  • [42] Disease Modeling of Severe Congenital Neutropenia Using CRISPR/Cas9 Gene Correction or Knockout of ELANE in Patients Derived Induced Pluripotent Stem Cells
    Nasri, Masoud
    Dannenmann, Benjamin
    Mir, Perihan
    Ritter, Malte U.
    Amend, Diana
    Xu, Yun
    Klimiankou, Maksim
    Zeidler, Cornelia
    Welte, Karl
    Skokowa, Julia
    BLOOD, 2019, 134
  • [43] Gene knockout in highly purified mouse hematopoietic stem cells by CRISPR/Cas9 technology
    Dong, Yan
    Bai, Haitao
    Dong, Fang
    Zhang, Xiao-Bing
    Ema, Hideo
    JOURNAL OF IMMUNOLOGICAL METHODS, 2021, 495
  • [44] Optimized CRISPR/Cas9 Editing Strategy for the Correction of CD40LG Gene in T Cells and Hematopoietic Stem Cells
    Vavassori, Valentina
    Mercuri, Elisabetta
    Schiroli, Giulia
    Marcovecchio, Genni
    Castiello, Maria Carmina
    Annoni, Andrea
    Albano, Luisa
    Capo, Valentina
    Margulies, Carrie
    Buquicchio, Frank
    Cotta-Ramusino, Cecilia
    Villa, Anna
    Naldini, Luigi
    Genovese, Pietro
    MOLECULAR THERAPY, 2019, 27 (04) : 308 - 309
  • [45] Editing the immune system in vivo in mice using CRISPR/Cas9 ribonucleoprotein (RNP)-mediated gene editing of transplanted hematopoietic stem cells
    Wang, Rui
    Graham, Sean
    Gao, Lei
    Tam, Jason
    Levesque, Marc C.
    METHODS, 2021, 194 : 30 - 36
  • [46] A Novel Anti-Cancer Therapy: CRISPR/Cas9 Gene Editing
    Chen, Xin-Zhu
    Guo, Rong
    Zhao, Cong
    Xu, Jing
    Song, Hang
    Yu, Hua
    Pilarsky, Christian
    Nainu, Firzan
    Li, Jing-Quan
    Zhou, Xin-Ke
    Zhang, Jian-Ye
    FRONTIERS IN PHARMACOLOGY, 2022, 13
  • [47] Genome and epigenome editing with CRISPR/Cas9 for gene therapy and disease modeling
    Gersbach, Charles A.
    TRANSGENIC RESEARCH, 2016, 25 (02) : 201 - 201
  • [48] Engineered Nanomaterials to Potentiate CRISPR/Cas9 Gene Editing for Cancer Therapy
    Yi, Ke
    Kong, Huimin
    Lao, Yeh-Hsing
    Li, Di
    Mintz, Rachel L.
    Fang, Tianxu
    Chen, Guojun
    Tao, Yu
    Li, Mingqiang
    Ding, Jianxun
    ADVANCED MATERIALS, 2024, 36 (13)
  • [49] Is microfluidics the "assembly line" for CRISPR-Cas9 gene-editing?
    Ahmadi, Fatemeh
    Quach, Angela B. V.
    Shih, Steve C. C.
    BIOMICROFLUIDICS, 2020, 14 (06)
  • [50] CRISPR/Cas9 Gene Editing of HeLa Cells to Tag Proteins with mNeonGreen
    Surve, Sachin
    Sorkin, Alexander
    BIO-PROTOCOL, 2022, 12 (10):
12345