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
  • [31] 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
  • [32] MILESTONE, A universal CRISPR/Cas9n-mediated genome editing strategy for ELANE-related severe congenital neutropenia
    Nasri, M.
    Ritter, M.
    Mir, P.
    Dannenmann, B.
    Kaufmann, M. M.
    Xu, Y.
    Borbaran-Bravo, N.
    Klimiankou, M.
    Lengerke, C.
    Zeidler, C.
    Cathomen, T.
    Welte, K.
    Skokowa, J.
    HUMAN GENE THERAPY, 2024, 35 (3-4) : A8 - A9
  • [33] Application of the CRISPR/Cas9-based Gene Editing Technology for the Treatment of NUT Carcinoma
    Choi, J.
    Yi, H. -L.
    Lee, M. -S.
    Kim, D.
    Choi, Y. -L.
    JOURNAL OF THORACIC ONCOLOGY, 2023, 18 (11) : S206 - S206
  • [34] Increasing the efficiency of CRISPR/Cas9-based gene editing by suppressing RNAi in plants
    Xiaoyan Wang
    Jiayun Lu
    Kangwen Lao
    Suikang Wang
    Xiaowei Mo
    Xintong Xu
    Xuemei Chen
    Beixin Mo
    Science China(Life Sciences), 2019, (07) : 982 - 984
  • [35] CRISPR/Cas9-based oncogene editing enables characterization of rare mutations in cancer cells
    Plenker, Dennis
    Sos, Martin L.
    CANCER RESEARCH, 2017, 77
  • [36] Genetic advancements in obesity management and CRISPR–Cas9-based gene editing system
    Muthukumaran Jayachandran
    Zhaoliang Fei
    Shen Qu
    Molecular and Cellular Biochemistry, 2023, 478 : 491 - 501
  • [37] Generation of a CRISPR/Cas9-Based Vector Specific for Gene Manipulation in Leishmania major
    Sangani, Ghodratollah Salehi
    Jajarmi, Vahid
    Khamesipour, Ali
    Mahmoudi, Mahmoud
    Fata, Abdolmajid
    Mohebali, Mehdi
    IRANIAN JOURNAL OF PARASITOLOGY, 2019, 14 (01) : 78 - 88
  • [38] Nucleic acid nanostructure for delivery of CRISPR/Cas9-based gene editing system
    Tang, Wantao
    Liu, Jianbing
    Ding, Baoquan
    INTERDISCIPLINARY MEDICINE, 2023, 1 (01):
  • [39] Increasing the efficiency of CRISPR/Cas9-based gene editing by suppressing RNAi in plants
    Xiaoyan Wang
    Jiayun Lu
    Kangwen Lao
    Suikang Wang
    Xiaowei Mo
    Xintong Xu
    Xuemei Chen
    Beixin Mo
    Science China Life Sciences, 2019, 62 : 982 - 984
  • [40] CRISPR/Cas9-based functional characterization of the pigmentation gene ebony in Plutella xylostella
    Xu, X.
    Harvey-Samuel, T.
    Yang, J.
    You, M.
    Alphey, L.
    INSECT MOLECULAR BIOLOGY, 2021, 30 (06) : 615 - 623
12345