A comparison of different strategies for CRISPR/Cas9-based gene therapies of congenital neutropenia associated with ELANE mutations

被引：0
|
作者：
Ritter, M. U. [1 ,2 ]
Nasri, M. [1 ,2 ]
Dannenmann, B. [1 ,2 ]
Mir, P. [2 ]
Secker, B. [2 ]
Zeidler, C. [3 ,4 ]
Klimiankou, M. [1 ,2 ,3 ,4 ]
Welte, K. [2 ,3 ,4 ]
Skokowa, J. [1 ,2 ,3 ,4 ]
机构：
[1] GRTC Tubingen, Tubingen, Germany
[2] Univ Hosp Tubingen, Tubingen, Germany
[3] Severe Congenital Neutropenia Int Registry Europe, Tubingen, Germany
[4] EuNet INNOCHRON, Iraklion, Greece
来源：
HUMAN GENE THERAPY | 2024年 / 35卷 / 3-4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：
P385
引用
收藏
页码：A175 / A175
页数：1
相关论文
共 50 条
  • [1] A Potential CRISPR/Cas9-Based Gene Therapy for Severe Congenital Neutropenia
    Ngoc Tung Tran
    Van Trung Chu
    Kuehn, Ralf
    Rajewsky, Klaus
    MOLECULAR THERAPY, 2019, 27 (04) : 309 - 310
  • [2] CRISPR-Cas9n-mediated ELANE promoter editing for gene therapy of severe congenital neutropenia
    Nasri, Masoud
    Ritter, Malte U.
    Mir, Perihan
    Dannenmann, Benjamin
    Kaufmann, Masako M.
    Arreba-Tutusaus, Patricia
    Xu, Yun
    Borbaran-Bravo, Natalia
    Klimiankou, Maksim
    Lengerke, Claudia
    Zeidler, Cornelia
    Cathomen, Toni
    Welte, Karl
    Skokowa, Julia
    MOLECULAR THERAPY, 2024, 32 (06) : 1628 - 1642
  • [3] CRISPR/Cas9 genome editing of hot-spot ELANE mutations in iPSCs of severe Congenital neutropenia patients
    Nasri, M.
    Dannenmann, B.
    Ritter, M.
    Xu, Y.
    Klimiankou, M.
    Zeidler, C.
    Lengerke, C.
    Welte, K.
    Skokowa, J.
    ONCOLOGY RESEARCH AND TREATMENT, 2021, 44 : 195 - 195
  • [4] Mutations in the ELANE Gene are Associated with Development of Periodontitis in Patients with Severe Congenital Neutropenia
    Ye, Ying
    Carlsson, Goran
    Wondimu, Biniyam
    Fahlen, Annika
    Karlsson-Sjoberg, Jenny
    Andersson, Mats
    Engstrand, Lars
    Yucel-Lindberg, Tulay
    Modeer, Thomas
    Putsep, Katrin
    JOURNAL OF CLINICAL IMMUNOLOGY, 2011, 31 (06) : 936 - 945
  • [5] Mutations in the ELANE Gene are Associated with Development of Periodontitis in Patients with Severe Congenital Neutropenia
    Ying Ye
    Göran Carlsson
    Biniyam Wondimu
    Annika Fahlén
    Jenny Karlsson-Sjöberg
    Mats Andersson
    Lars Engstrand
    Tülay Yucel-Lindberg
    Thomas Modéer
    Katrin Pütsep
    Journal of Clinical Immunology, 2011, 31 : 936 - 945
  • [6] Optimizing CRISPR/Cas9-based gene manipulation in echinoderms
    Oulhen, Nathalie
    Pieplow, Cosmo
    Perillo, Margherita
    Gregory, Pauline
    Wessel, Gary M.
    DEVELOPMENTAL BIOLOGY, 2022, 490 : 117 - 124
  • [7] Efficient CRISPR/Cas9-based gene knockout in watermelon
    Shouwei Tian
    Linjian Jiang
    Qiang Gao
    Jie Zhang
    Mei Zong
    Haiying Zhang
    Yi Ren
    Shaogui Guo
    Guoyi Gong
    Fan Liu
    Yong Xu
    Plant Cell Reports, 2017, 36 : 399 - 406
  • [8] Efficient CRISPR/Cas9-based gene knockout in watermelon
    Tian, Shouwei
    Jiang, Linjian
    Gao, Qiang
    Zhang, Jie
    Zong, Mei
    Zhang, Haiying
    Ren, Yi
    Guo, Shaogui
    Gong, Guoyi
    Liu, Fan
    Xu, Yong
    PLANT CELL REPORTS, 2017, 36 (03) : 399 - 406
  • [9] The use of CRISPR/Cas9-based gene editing strategies to explore cancer gene function in mice
    van der Weyden, Louise
    Jonkers, Jos
    Adams, David J.
    CURRENT OPINION IN GENETICS & DEVELOPMENT, 2021, 66 : 57 - 62
  • [10] CRISPR/Cas9-based Gene Therapies for Fighting Drug Resistance Mediated by Cancer Stem Cells
    Dashtaki, Masoumeh Eliyasi
    Ghasemi, Sorayya
    CURRENT GENE THERAPY, 2023, 23 (01) : 41 - 50
12345