Preclinical development of a TALEN®-based genome editing therapy for RAG1 deficiency

被引：0
|
作者：
Rhiel, M. [1 ,2 ]
Klermund, J. [1 ,2 ]
Jullierat, A. [3 ]
Andrieux, G. [4 ,5 ,6 ]
Rositzka, J. [1 ,2 ]
Duchateau, P. [7 ]
Cathomen, T. [1 ,2 ]
Cornu, T. I. [1 ,2 ]
机构：
[1] Univ Freiburg, Inst Transfus Med & Gene Therapy, Med Ctr, D-79106 Freiburg, Germany
[2] Univ Freiburg, Ctr Chron Immunodeficiency CCI, Med Ctr, D-79106 Freiburg, Germany
[3] Cellectis Inc, New York, NY 10016 USA
[4] Univ Freiburg, Inst Med Bioinformat & Syst Med, Med Ctr, D-79110 Freiburg, Germany
[5] German Canc Consortium DKTK, D-79106 Freiburg, Germany
[6] Partner Site Freiburg, German Canc Res Ctr DKFZ, D-79106 Freiburg, Germany
[7] Cellectis SA, F-75013 Paris, France
来源：
HUMAN GENE THERAPY | 2021年 / 32卷 / 19-20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：
OR34
引用
收藏
页码：A13 / A14
页数：2
相关论文
共 50 条
  • [31] A phylogeny of putative perciform suborders based on the nuclear RAG1 gene
    Mahon, AR
    Carpenter, KE
    INTEGRATIVE AND COMPARATIVE BIOLOGY, 2004, 44 (06) : 723 - 723
  • [32] RAG1 and RAG2 expression in normal mature B lymphocytes in humans; Implications for receptor editing in prevention of autoimmune responses.
    Miura, K
    Suzuki, N
    Takeba, Y
    Ohwada, S
    Isida, M
    Sakane, T
    ARTHRITIS AND RHEUMATISM, 1998, 41 (09): : S285 - S285
  • [33] A Genomic Editing-Based Therapeutic Approach for RAG2 Deficiency
    Pavel-Dinu, Mara
    Gardner, Cameron
    La Guardia, Thomas A.
    Vakulskas, Christopher A.
    Lee, Ciaran
    Bao, Gang
    Sheikali, Adam
    Menezes, Talia
    Notarangelo, Luigi D.
    Porteus, Matthew H.
    MOLECULAR THERAPY, 2020, 28 (04) : 55 - 56
  • [34] Basics and practices of genome editing-examples from development to application of TALEN and CRISPR systems
    Sakuma, Tetsushi
    JOURNAL OF PHARMACOLOGICAL SCIENCES, 2016, 130 (03) : S18 - S18
  • [35] Hypomorphic Rag1 mutations alter the preimmune repertoire at early stages of lymphoid development
    de Bruin, L. M. Ott
    Bosticardo, M.
    Barbieri, A.
    Lin, S. G.
    Rowe, J. H.
    Poliani, P. L.
    Ching, K.
    Eriksson, D.
    Landegren, N.
    Kampe, O.
    Manis, J. P.
    Notarangelo, L. D.
    BLOOD, 2018, 132 (03) : 281 - 292
  • [36] Diagnostic approach in a young adult with RAG1 variants initially diagnosed with specific antibody deficiency and bronchiectasis
    Franson, Brady
    Potts, David Evan
    Ujhazi, Boglarka
    Miller, Rahim
    Blair, Grace
    Toth, Marta
    Wang, Chen
    Petrov, Andrej
    Walter, Jolan
    CLINICAL IMMUNOLOGY, 2024, 262
  • [37] The roles of the RAG1 and RAG2 “non-core” regions in V(D)J recombination and lymphocyte development
    Jessica M. Jones
    Carrie Simkus
    Archivum Immunologiae et Therapiae Experimentalis, 2009, 57 : 105 - 116
  • [38] Reconstruction of polyreactive B-cell lineages helps understanding autoimmunity in RAG1/2 deficiency
    Blazso, Peter
    Csomos, Krisztian
    Ujhazi, Boglarka
    Walter, Jolan E.
    EUROPEAN JOURNAL OF IMMUNOLOGY, 2023, 53 : 46 - 46
  • [39] Generation of inactivated IL2RG and RAG1 monkeys with severe combined immunodeficiency using base editing
    Zheng, Xiao
    Huang, Chunhui
    Lin, Yingqi
    Han, Bofeng
    Chen, Yizhi
    Li, Caijuan
    Li, Jiawei
    Ding, Yongyan
    Song, Xichen
    Wang, Wei
    Liang, Weien
    Wu, Jianhao
    Wu, Jiaxi
    Gao, Jiale
    Wei, Chengxi
    Zhang, Xudong
    Tu, Zhuchi
    Yan, Sen
    SIGNAL TRANSDUCTION AND TARGETED THERAPY, 2023, 8 (01)
  • [40] Soybean Aphid (Hemiptera: Aphididae) Development on Soybean With Rag1 Alone, Rag2 Alone, and Both Genes Combined
    Wiarda, S. L.
    Fehr, W. R.
    O'Neal, M. E.
    JOURNAL OF ECONOMIC ENTOMOLOGY, 2012, 105 (01) : 252 - 258
12345