20 years of gene therapy for SCID

被引:107
作者
Fischer, Alain [1 ,2 ,3 ]
Hacein-Bey-Abina, Salima [1 ,2 ,4 ]
Cavazzana-Calvo, Marina [1 ,2 ,4 ]
机构
[1] INSERM, Paris, France
[2] Paris Descartes Univ, Paris, France
[3] Hop Necker Enfants Malad, AP HP, Unite Immunol & Hematol Pediat, Paris, France
[4] Hop Necker Enfants Malad, AP HP, Ctr Invest Clin Integre Biotherapies, Dept Biotherapy, Paris, France
关键词
SEVERE COMBINED IMMUNODEFICIENCY; LENTIVIRAL VECTOR; CANCER; DISEASE; TRIALS; RECONSTITUTION; LYMPHOCYTES; DEFICIENCY; DOUBTS; CELLS;
D O I
10.1038/ni0610-457
中图分类号
R392 [医学免疫学]; Q939.91 [免疫学];
学科分类号
100102 ;
摘要
Severe combined immunodeficiency conditions are devastating disorders of adaptive immunity. Although these diseases were initially treated by transplantation of allogeneic hematopoietic stem cells, the past 20 years has shown that these conditions are correctable by gene therapy.
引用
收藏
页码:457 / 460
页数:4
相关论文
共 30 条
[1]   Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning [J].
Aiuti, A ;
Slavin, S ;
Aker, M ;
Ficara, F ;
Deola, S ;
Mortellaro, A ;
Morecki, S ;
Andolfi, G ;
Tabucchi, A ;
Carlucci, F ;
Marinello, E ;
Cattaneo, F ;
Vai, S ;
Servida, P ;
Miniero, R ;
Roncarolo, MG ;
Bordignon, C .
SCIENCE, 2002, 296 (5577) :2410-2413
[2]   Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency. [J].
Aiuti, Alessandro ;
Cattaneo, Federica ;
Galimberti, Stefania ;
Benninghoff, Ulrike ;
Cassani, Barbara ;
Callegaro, Luciano ;
Scaramuzza, Samantha ;
Andolfi, Grazia ;
Mirolo, Massimiliano ;
Brigida, Immacolata ;
Tabucchi, Antonella ;
Carlucci, Filippo ;
Eibl, Martha ;
Aker, Memet ;
Slavin, Shimon ;
Al-Mousa, Hamoud ;
Al Ghonaium, Abdulaziz ;
Ferster, Alina ;
Duppenthaler, Andrea ;
Notarangelo, Luigi ;
Wintergerst, Uwe ;
Buckley, Rebecca H. ;
Bregni, Marco ;
Marktel, Sarah ;
Valsecchi, Maria Grazia ;
Rossi, Paolo ;
Ciceri, Fabio ;
Miniero, Roberto ;
Bordignon, Claudio ;
Roncarolo, Maria-Grazia .
NEW ENGLAND JOURNAL OF MEDICINE, 2009, 360 (05) :447-458
[3]   T-LYMPHOCYTE-DIRECTED GENE-THERAPY FOR ADA(-) SCID - INITIAL TRIAL RESULTS AFTER 4 YEARS [J].
BLAESE, RM ;
CULVER, KW ;
MILLER, AD ;
CARTER, CS ;
FLEISHER, T ;
CLERICI, M ;
SHEARER, G ;
CHANG, L ;
CHIANG, YW ;
TOLSTOSHEV, P ;
GREENBLATT, JJ ;
ROSENBERG, SA ;
KLEIN, H ;
BERGER, M ;
MULLEN, CA ;
RAMSEY, WJ ;
MUUL, L ;
MORGAN, RA ;
ANDERSON, WF .
SCIENCE, 1995, 270 (5235) :475-480
[4]   GENE-THERAPY IN PERIPHERAL-BLOOD LYMPHOCYTES AND BONE-MARROW FOR ADA(-) IMMUNODEFICIENT PATIENTS [J].
BORDIGNON, C ;
NOTARANGELO, LD ;
NOBILI, N ;
FERRARI, G ;
CASORATI, G ;
PANINA, P ;
MAZZOLARI, E ;
MAGGIONI, D ;
ROSSI, C ;
SERVIDA, P ;
UGAZIO, AG ;
MAVILIO, F .
SCIENCE, 1995, 270 (5235) :470-475
[5]   Diversity, functionality, and stability of the T cell repertoire derived in vivo from a single human T cell precursor [J].
Bousso, P ;
Wahn, V ;
Douagi, I ;
Horneff, G ;
Pannetier, C ;
Le Deist, F ;
Zepp, F ;
Niehues, T ;
Kourilsky, P ;
Fischer, A ;
de Saint Basile, G .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2000, 97 (01) :274-278
[6]   Molecular defects in human severe combined immunodeficiency and approaches to immune reconstitution [J].
Buckley, RH .
ANNUAL REVIEW OF IMMUNOLOGY, 2004, 22 :625-655
[7]   Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy [J].
Cartier, Nathalie ;
Hacein-Bey-Abina, Salima ;
Bartholomae, Cynthia C. ;
Veres, Gabor ;
Schmidt, Manfred ;
Kutschera, Ina ;
Vidaud, Michel ;
Abel, Ulrich ;
Dal-Cortivo, Liliane ;
Caccavelli, Laure ;
Mahlaoui, Nizar ;
Kiermer, Veronique ;
Mittelstaedt, Denice ;
Bellesme, Celine ;
Lahlou, Najiba ;
Lefrere, Francois ;
Blanche, Stephane ;
Audit, Muriel ;
Payen, Emmanuel ;
Leboulch, Philippe ;
l'Homme, Bruno ;
Bougneres, Pierre ;
Von Kalle, Christof ;
Fischer, Alain ;
Cavazzana-Calvo, Marina ;
Aubourg, Patrick .
SCIENCE, 2009, 326 (5954) :818-823
[8]   Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease [J].
Cavazzana-Calvo, M ;
Hacein-Bey, S ;
Basile, CD ;
Gross, F ;
Yvon, E ;
Nusbaum, P ;
Selz, F ;
Hue, C ;
Certain, S ;
Casanova, JL ;
Bousso, P ;
Le Deist, F ;
Fischer, A .
SCIENCE, 2000, 288 (5466) :669-672
[9]   Gene therapy: A tragic setback [J].
Check, E .
NATURE, 2002, 420 (6912) :116-118
[10]   Gene therapy put on hold as third child develops cancer [J].
Check, E .
NATURE, 2005, 433 (7026) :561-561