Preclinical biosafety studies of lentiviral vector-mediated gene therapy in erythrocyte pyruvate kinase deficiency

被引：0
|
作者：
Quintana-Bustamante, O. [1 ]
Sanchez-Dominguez, R. [1 ]
Garcia-Torralba, A. [1 ]
Alberquilla, O. [1 ]
Lopez-Manzaneda, S. [1 ]
Fananas, S. [1 ]
Nieto, V. [1 ]
Molinos, A. [1 ]
Garcia-Bravo, M. [1 ]
Yanez, R. [1 ]
Bueren, J. A. [1 ]
Navarro, S. [1 ]
Segovia, J. C. [1 ]
机构：
[1] FJD, IIS, CIBERER, CIEMAT, Madrid, Spain
来源：
HUMAN GENE THERAPY | 2018年 / 29卷 / 12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：
P031
引用
收藏
页码：A39 / A39
页数：1
相关论文
共 50 条
  • [1] Oncoretroviral and lentiviral vector-mediated gene therapy
    VandenDriessche, T
    Naldini, L
    Collen, D
    Chuah, MKL
    GENE THERAPY METHODS, 2002, 346 : 573 - 589
  • [2] Development and Validation of a GMP Grade Lentiviral Vector for the Gene Therapy of Pyruvate Kinase Deficiency
    Navarro, Susana
    Trigueros, Cesar
    Quintana-Bustamante, Oscar
    Villanueva, Mariela S.
    Lopez-Manzaneda, Sergio
    Bueren, Juan A.
    Ramirez, Juan C.
    Segovia, Jose C.
    MOLECULAR THERAPY, 2017, 25 (05) : 126 - 126
  • [3] Preclinical lentiviral vector-mediated gene transfer for RAG1 and RAG2 deficiency
    Pike-Overzet, Karin
    Rodijk, Mark
    Ng, Yuk-Yin
    Baert, Miranda R. M.
    Schambach, Axel
    Baum, Christopher
    Wagemaker, Gerard
    van Dongen, Jacques J. M.
    Staal, Frank J. T.
    HUMAN GENE THERAPY, 2007, 18 (10) : 1011 - 1011
  • [4] Preclinical studies of efficacy thresholds and tolerability of a clinically ready lentiviral vector for pyruvate kinase deficiency treatment
    Navarro, Susana
    Quintana-Bustamante, Oscar
    Sanchez-Dominguez, Rebeca
    Lopez-Manzaneda, Sergio
    Ojeda-Perez, Isabel
    Garcia-Torralba, Aida
    Alberquilla, Omaira
    Law, Kenneth
    Beard, Brian C.
    Bastone, Antonella
    Rothe, Michael
    Villanueva, Mariela
    Ramirez, Juan C.
    Fananas-Baquero, Sara
    Nieto-Romero, Virginia
    Molinos-Vicente, Andrea
    Gutierrez, Sonia
    Nicoletti, Eileen
    Garcia-Bravo, Maria
    Bueren, Juan A.
    Schwartz, Jonathan D.
    Segovia, Jose-Carlos
    MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2021, 22 : 350 - 359
  • [5] Lentiviral-based gene therapy for Pyruvate Kinase deficiency
    Garcia-Gomez, M.
    Navarro, S.
    Orman, I.
    Angel Martin, M.
    Garcia-Bravo, M.
    Bueren, J. A.
    Antoniou, M.
    Segovia, J. C.
    HUMAN GENE THERAPY, 2013, 24 (12) : A111 - A111
  • [6] Gene therapy of the human erythrocyte pyruvate kinase deficiency.
    Segovia, JC
    Meza, NW
    Quintana-Bustamante, O
    Puyet, A
    Rio, P
    Bueren, JA
    Bautista, JM
    BLOOD, 2004, 104 (11) : 454A - 454A
  • [7] Efficient gene therapy for the treatment of erythrocyte pyruvate kinase deficiency
    Meza, Nestor W.
    Alonso, Maria E.
    Navarro, Susana
    Guenechea, Guillermo
    Quintana-Bustamante, Oscar
    Garcia-Gomez, Mara
    Bueren, Juan A.
    Bautista, Jose M.
    Segovia, Jose C.
    HUMAN GENE THERAPY, 2007, 18 (10) : 959 - 959
  • [8] Global phase 1 study results of lentiviral mediated gene therapy for severe pyruvate kinase deficiency
    Sevilla, J.
    Shah, A. J.
    Lorenzo, J. L. L.
    Navarro, S.
    Lanos, L.
    de Camino Gaisse, B. P.
    Sanchez, S.
    Zubicaray, J.
    Glader, B.
    Chien, M.
    Bustamante, O. Q.
    Zeini, M.
    Choi, G.
    Nicoletti, E.
    Rao, G. R.
    Roncarolo, M. G.
    Bueren, J. A.
    Schwartz, J. D.
    Segovia, J. C.
    HUMAN GENE THERAPY, 2024, 35 (3-4) : A4 - A4
  • [9] Global Phase 1 Study Results of Lentiviral Mediated Gene Therapy for Severe Pyruvate Kinase Deficiency
    Shah, Ami J.
    Lopez Lorenzo, Jose Luis
    Sevilla, Julian
    Navarro, Susana
    Llanos, Lucia
    de Camino Gaisse, Begona Perez
    Sanchez, Sol
    Zubicaray, Josune
    Glader, Bertil
    Chien, May
    Quintana Bustamante, Oscar
    Zeini, Miriam
    Choi, Grace
    Nicoletti, Eileen
    Rao, Gayatri
    Roncarolo, Maria-Grazia
    Bueren, Juan A.
    Schwartz, Jonathan
    Carlos Segovia, Jose
    MOLECULAR THERAPY, 2023, 31 (04) : 118 - 119
  • [10] Lentiviral vector-mediated gene therapy for a mouse model of spinal muscular atrophy
    Azzouz, M
    Le, TN
    Waddington, S
    Walmsley, L
    Monani, U
    Wilkes, FJ
    Themis, M
    Mitrophanous, KA
    Burghes, A
    Mazarakis, ND
    MOLECULAR THERAPY, 2003, 7 (05) : S248 - S248
12345