An in vitro Comparison of known Gene Therapy Approaches for Congenital Neutropenia-inducing ELANE Mutations

被引:0
|
作者
Ritter, M. U. [1 ]
Nasri, M. [1 ]
Dannenmann, B. [1 ]
Mir, P. [1 ]
Secker, B. [1 ]
Zeidler, C. [2 ]
Klimiankou, M. [1 ]
Welte, K. [3 ]
Skokowa, J. [1 ]
机构
[1] Univ Klinkum Tubingen, Innere Med 2, Tubingen, Germany
[2] Hannover Med Sch, Zentrum Seltene Erkrankungen, Hannover, Germany
[3] Univ Tubingen, Kinderheilkunde 1, Tubingen, Germany
关键词
D O I
暂无
中图分类号
R73 [肿瘤学];
学科分类号
100214 ;
摘要
V138
引用
收藏
页码:40 / 40
页数:1
相关论文
共 50 条
  • [41] Mutations in the neutrophil elastase gene in cyclic and congenital neutropenia
    Aprikyan, AAG
    Dale, DC
    CURRENT OPINION IN IMMUNOLOGY, 2001, 13 (05) : 535 - 538
  • [42] ELANE mutations causing severe congenital neutropenia in humans, but induce only a weak neutropenic phenotype in mouse.
    Kanoza, Michal
    Kwiecinski, Jakub
    Ciaston, Izabela
    Potempa, Jan
    Koziel, Joanna
    EUROPEAN JOURNAL OF IMMUNOLOGY, 2023, 53 : 127 - 127
  • [43] Start Codon ELANE Mutations are Necessary and Sufficient to Induce the Promyelocyte Differentiation Block Observed in Severe Congenital Neutropenia
    Trump, Lisa
    Nayak, Ramesh
    Singh, Abishek
    Emberesh, Sana
    Myers, Kasiani
    Horwitz, Marshall
    Lutzko, Carolyn
    Cancelas, Jose A.
    MOLECULAR THERAPY, 2019, 27 (04) : 398 - 398
  • [44] Mutations in the gene encoding neutrophil elastase in congenital and cyclic neutropenia
    Dale, DC
    Person, RE
    Bolyard, AA
    Aprikyan, AG
    Bos, C
    Bonilla, MA
    Boxer, LA
    Kannourakis, G
    Zeidler, C
    Welte, K
    Benson, KF
    Horwitz, M
    BLOOD, 2000, 96 (07) : 2317 - 2322
  • [45] Efficient, Specific and Universal Therapeutic Gene Editing of ELANE for Severe Congenital Neutropenia in Human Hematopoietic Stem Cells
    Rao, Shuquan
    Brito-Frazao, Josias
    Yao, Qiuming
    Serbin, Anna V.
    Luk, Kevin
    Wu, Yuxuan
    Zeng, Jing
    Ren, Chunyan
    Shen, Anne H.
    Watkinson, Ruth
    Armant, Myriam
    Pinello, Luca
    Wolfe, Scot A.
    Lee, Benhur
    Chiarle, Roberto
    Shimamura, Akiko
    Newburger, Peter
    Bauer, Daniel E.
    MOLECULAR THERAPY, 2020, 28 (04) : 55 - 55
  • [46] Double de novo mutations of ELANE (ELA2) in a patient with severe congenital neutropenia requiring high-dose G-CSF therapy
    Lunden, Lina
    Boxhammer, Sandra
    Carlsson, Goran
    Ellstrom, Karl-Gustav
    Nordenskjold, Magnus
    Lagerstedt-Robinson, Kristina
    Fadeel, Bengt
    BRITISH JOURNAL OF HAEMATOLOGY, 2009, 147 (04) : 587 - 590
  • [47] 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
  • [48] The causal role of mutations in the neutrophil elastase gene in cyclic and congenital neutropenia
    Aprikyan, A
    Stein, S
    Kutyavin, T
    Rodger, E
    Bolyard, A
    Dale, D
    EXPERIMENTAL HEMATOLOGY, 2002, 30 (06) : 89 - 89
  • [49] Clinical Characteristics and Treatment Response of a Novel ELANE Gene Mutation (c.295_303del) in Congenital Neutropenia
    Ning, Junjie
    MEDITERRANEAN JOURNAL OF HEMATOLOGY AND INFECTIOUS DISEASES, 2024, 16 (01)
  • [50] Case Report: Characterization of known (c.607G>C) and novel (c.416C>G) ELANE mutations in two Mexican families with congenital neutropenia
    Nunez-Nunez, Maria Enriqueta
    Lona-Reyes, Juan Carlos
    Lopez-Barragan, Brenda
    Cruz-Osorio, Rosa Margarita
    Gutierrez-Zepeda, Bricia Melissa
    Quintero-Ramos, Antonio
    Becerra-Loaiza, Denisse Stephania
    FRONTIERS IN IMMUNOLOGY, 2023, 14