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
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
  • [31] Lentiviral vector-mediated gene delivery into human embryonic stem cells
    Gropp, Michal
    Reubinoff, Benjamin
    STEM CELL TOOLS AND OTHER EXPERIMENTAL PROTOCOLS, 2006, 420 : 64 - 81
  • [32] OPTIMIZATION OF TRANSDUCTION CONDITIONS WITH GMP LIKE LENTIVIRAL VECTORS FOR THE GENE THERAPY OF PYRUVATE KINASE DEFICIENCY
    Navarro, S.
    Quintana-Bustamante, O.
    Trigueros, C.
    Villanueva, M.
    Lopez-Manzaneda, S.
    Bueren, J. A.
    Ramirez, J. C.
    Segovia, J. C.
    HAEMATOLOGICA, 2017, 102 : 451 - 452
  • [33] Development of Tissue-Specific Lentiviral Vectors for the Gene Therapy of the Erythroid Pyruvate Kinase Deficiency
    Garcia-Gomez, M.
    Meza, N. W.
    Navarro, S.
    Alonso-Ferrero, M. E.
    Bueren, J. A.
    Segovia, J. C.
    HUMAN GENE THERAPY, 2009, 20 (09) : 1091 - 1091
  • [34] Tissue-Specific Lentiviral Vectors as a Tool for the Gene Therapy of the Erythroid Pyruvate Kinase Deficiency
    Garcia-Gomez, Maria
    Meza, Nestor W.
    Navarro, Susana
    Alonso-Ferrero, Maria E.
    Bueren, Juan A.
    Segovia, Jose C.
    MOLECULAR THERAPY, 2009, 17 : S119 - S120
  • [35] Lentiviral Vector-Mediated Gene Complementation for the Diagnosis of LAD-I
    Mesa-Nunez, Cristina
    Damian, Carlos
    Gilmour, Kimberly
    Sanchez, Rebeca
    Alberquilla, Omaira
    Segovia, Jose C.
    Booth, Claire
    Bueren, Juan A.
    Ip, Winnie
    Almarza, Elena
    MOLECULAR THERAPY, 2020, 28 (04) : 176 - 177
  • [36] Characterization of lentiviral vector-mediated gene transfer in adult mouse brain
    Baekelandt, V
    Claeys, A
    Eggermont, K
    Lauwers, E
    De Strooper, B
    Nuttin, B
    Debyser, Z
    HUMAN GENE THERAPY, 2002, 13 (07) : 841 - 853
  • [37] Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: Interim Results of a Global Phase 1 Study for Adult and Pediatric Patients
    Shah, Ami J.
    Lopez Lorenzo, Jose Luis
    Navarro, Susana
    Sevilla, Julian
    Llanos, Lucia
    Perez de Camino Gaisse, Begona
    Sanchez, Sol
    Glader, Bert
    Chien, May
    Quintana Bustamante, Oscar
    Beard, Brian C.
    Law, Kenneth M.
    Zeini, Miriam
    Choi, Grace
    Nicoletti, Eileen
    Rao, Gayatri R.
    Grazia Roncarolo, Maria
    Bueren, Juan A.
    Schwartz, Jonathan D.
    Segovia, Jose C.
    BLOOD, 2021, 138
  • [38] Lentiviral mediated gene therapy for pyruvate kinase deficiency: Interim results of a global phase 1 study for adult and pediatric patients
    Lorenzo, J. L. Lopez
    Shah, A. J.
    Navarro, S.
    Sevilla, J.
    Llanos, L.
    Gaisse, B. P. de Camino
    Sanchez, S.
    Glader, B.
    Chien, M.
    Bustamante, O. Quintana
    Beard, B. C.
    Zeini, M.
    Choi, G.
    Nicoletti, E.
    Rao, G. R.
    Roncarolo, M. G.
    Bueren, J. A.
    Schwartz, J.
    Segovia, J. C.
    HUMAN GENE THERAPY, 2021, 32 (19-20) : A25 - A25
  • [39] Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: Updated Results of a Global Phase 1 Study for Adult and Pediatric Patients
    Lopez Lorenzo, Jose Luis
    Shah, Ami J.
    Navarro, Susana
    Sevilla, Julian
    Llanos, Lucia
    Perez Camino de Gaisse, Begona
    Sanchez, Sol
    Glader, Bertil
    Chien, May
    Quintana-Bustamante, Oscar
    Beard, Brian C.
    Law, Kenneth M.
    Zeini, Miriam
    Choi, Grace
    Nicoletti, Eileen
    Rao, Gayatri R.
    Roncarolo, Maria Grazia
    Bueren, Juan A.
    Schwartz, Jonathan D.
    Segovia, Jose C.
    MOLECULAR THERAPY, 2021, 29 (04) : 42 - 43
  • [40] LENTIVIRAL-MEDIATED GENE THERAPY FOR SEVERE PYRUVATE KINASE DEFICIENCY: RESULTS FROM AN ONGOING GLOBAL PHASE 1 STUDY
    Lopez Lorenzo, Jose Luis
    Shah, Ami
    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
    Schwartz, Jonathan
    Carlos Segovia, Jose
    BONE MARROW TRANSPLANTATION, 2023, 58 (SUPP1) : 275 - 276