Self-Inactivating Lentiviral Vectors for Correction of Rag1 Severe Combined Immunodeficiency

被引：0

作者：

Pike-Overzet, K. ^{[1
]}

Rodijk, M. ^{[1
]}

Ng, Y. Y. ^{[1
]}

Baert, M. R. M. ^{[1
]}

Lagresle-Peyrou, C. ^{[2
,3
]}

Schambach, A. ^{[4
]}

Zhang, F. ^{[5
]}

Hoeben, R. C. ^{[6
]}

Hacein-Bey-Abina, S. ^{[2
,3
]}

Lankester, A. C. ^{[7
]}

Bredius, R. G. M. ^{[7
]}

Driessen, G. J. A. ^{[8
]}

Thrasher, A. J. ^{[5
]}

Baum, C. ^{[4
]}

Cavazzana-Calvo, M. ^{[2
,3
]}

van Dongen, J. J. M. ^{[9
]}

Staal, F. J. T. ^{[1
]}

机构：

[1] Leiden Univ, Med Ctr, Dept Immunohematol & Blood Transfus, NL-2333 ZA Leiden, Netherlands

[2] Hop Necker Enfants Malad, INSERM, Paris, France

[3] Hop Necker Enfants Malad, Dept Biotherapy, Ctr Invest Clin Integre Biotherapies, Paris, France

[4] Hannover Med Sch, Inst Expt Hematol, D-3000 Hannover, Germany

[5] UCL, Inst Child Hlth, Mol Immunol Unit, London, England

[6] Leiden Univ, Med Ctr, Dept Mol Cell Biol, Leiden, Netherlands

[7] Leiden Univ, Med Ctr, Dept Pediat Immunol Hematooncol Bone Marrow Trans, Leiden, Netherlands

[8] Erasmus MC Univ, Med Ctr, Dept Pediat, Rotterdam, Netherlands

[9] Erasmus Univ, Med Ctr, Dept Immunol, Rotterdam, Netherlands

来源：

HUMAN GENE THERAPY | 2012年 / 23卷 / 10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：

引用

页码：A42 / A42

页数：1

共 50 条

[1] SELF-INACTIVATING LENTIVIRAL VECTORS FOR CORRECTION OF RAG1 SEVERE COMBINED IMMUNODEFICIENCY
Pike-Overzet, Karin
Rodijk, Mark
Ng, Yuk Yin
Baert, Miranda
Lagresle-Peyrou, Chantal
Schambach, Axel
Zhang, Fang
Hoeben, Rob
Hacein-Bey-Abina, Salima
Lankester, Arjan
Bredius, Robbert
Driessen, Gertjan
Thrasher, Adrian
Baum, Christopher
Cavazzana-Calvo, Marina
van Dongen, Jacques
Staal, Frank
EXPERIMENTAL HEMATOLOGY, 2012, 40 (08) : S46 - S47
[2] Self-Inactivating Lentiviral Vectors for Correction of Rag1 Severe Combined Immunodeficiency
Pike-Overzet, Karin
Wiekmeijer, Anna-Sophia
Baert, Miranda R. M.
Lagresle-Peyrou, Chantal
Schambach, Axel
Zhang, Fang
Hoeben, Rob C.
Hacein-Bey-Abina, Salima
Lankester, Arjen C.
Bredius, Robbert G. M.
Driessen, Gertjan J. A.
Thrasher, Adrian J.
Baum, Christopher
Cavazzana-Calvo, Marina
van Dongen, Jacques J. M.
Staal, Frank J. T.
MOLECULAR THERAPY, 2013, 21 : S120 - S121
[3] Correction of murine Rag1 deficiency by self-inactivating lentiviral vector-mediated gene transfer
K Pike-Overzet
M Rodijk
Y-Y Ng
M R M Baert
C Lagresle-Peyrou
A Schambach
F Zhang
R C Hoeben
S Hacein-Bey-Abina
A C Lankester
R G M Bredius
G J A Driessen
A J Thrasher
C Baum
M Cavazzana-Calvo
J J M van Dongen
F J T Staal
Leukemia, 2011, 25 : 1471 - 1483
[4] Correction of murine Rag1 deficiency by self-inactivating lentiviral vector-mediated gene transfer
Pike-Overzet, K.
Rodijk, M.
Ng, Y-Y
Baert, M. R. M.
Lagresle-Peyrou, C.
Schambach, A.
Zhang, F.
Hoeben, R. C.
Hacein-Bey-Abina, S.
Lankester, A. C.
Bredius, R. G. M.
Driessen, G. J. A.
Thrasher, A. J.
Baum, C.
Cavazzana-Calvo, M.
van Dongen, J. J. M.
Staal, F. J. T.
LEUKEMIA, 2011, 25 (09) : 1471 - 1483
[5] Finding levels of RAG1 expression that will correct RAG1 Severe Combined Immunodeficiency
Pike-Overzet, K.
Baert, M. R. M.
Schambach, A.
Rothe, M.
Zhang, F.
Lankester, A. C.
Bredius, R. G. M.
Thrasher, A. J.
Gaspar, H. B.
Staal, F. J. T.
HUMAN GENE THERAPY, 2014, 25 (11) : A41 - A41
[6] Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency
Thornhill, Susannah I.
Schambach, Axel
Howe, Steven J.
Ulaganathan, Meera
Grassman, Elke
Williams, David
Schiedlmeier, Bernhard
Sebire, Neil J.
Gaspar, H. Bobby
Kinnon, Christine
Baum, Christopher
Thrasher, Adrian J.
MOLECULAR THERAPY, 2008, 16 (03) : 590 - 598
[7] Uncovering and Dissecting the Genotoxicity of Self-inactivating Lentiviral Vectors In Vivo
Cesana, Daniela
Ranzani, Marco
Volpin, Monica
Bartholomae, Cynthia
Duros, Caroline
Artus, Alexandre
Merella, Stefania
Benedicenti, Fabrizio
Sergi, Lucia Sergi
Sanvito, Francesca
Brombin, Chiara
Nonis, Alessandro
Di Serio, Clelia
Doglioni, Claudio
von Kalle, Christof
Schmidt, Manfred
Cohen-Haguenauer, Odile
Naldini, Luigi
Montini, Eugenio
MOLECULAR THERAPY, 2014, 22 (04) : 774 - 785
[8] Mobilization and mechanism of transcription of integrated self-inactivating lentiviral vectors
Hanawa, H
Persons, DA
Nienhuis, AW
JOURNAL OF VIROLOGY, 2005, 79 (13) : 8410 - 8421
[9] RAG1/2 Knockout Pigs with Severe Combined Immunodeficiency
Huang, Jiao
Guo, Xiaogang
Fan, Nana
Song, Jun
Zhao, Bentian
Ouyang, Zhen
Liu, Zhaoming
Zhao, Yu
Yan, Quanmei
Yi, Xiaoling
Schambach, Axel
Frampton, Jon
Esteban, Miguel A.
Yang, Dongshan
Yang, Huaqiang
Lai, Liangxue
JOURNAL OF IMMUNOLOGY, 2014, 193 (03): : 1496 - 1503
[10] Novel RAG1 mutation in a case of severe combined immunodeficiency
Zhang, JY
Quintal, L
Atkinson, A
Williams, B
Grunebaum, E
Roifman, CM
PEDIATRICS, 2005, 116 (03) : E445 - E449

← 1 2 3 4 5 →