Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

Journal of Clinical Investigation - Tập 118 Số 9 - Trang 3132-3142 - 2008
Salima Hacein‐Bey‐Abina1, Alexandrine Garrigue2, Gary P. Wang3, Jean Soulier4, Apiradee Lim5, Estelle Morillon2, Emmanuelle Clappier5, Laure Caccavelli6, Éric Delabesse7, Kheïra Beldjord8,9, Vahid Asnafi8,9, Elizabeth Macintyre8,9, Liliane Dal Cortivo6, Isabelle Radford9, Nicole Brousse10, François Sigaux4, Despina Moshous11, Julia Hauer2, Arndt Borkhardt12, Bernd H. Belohradsky13, Uwe Wintergerst13, María Velez14, Lily E. Leiva14, Ricardo U. Sorensen14, N. M. Wulffraat15, Stephan Ehl11, Frederic D. Bushman3, Alain Fischer11,2, Marina Cavazzana6,2
1Department of Biotherapy, Hôpital Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (AP-HP), Université René Descartes, Paris, France. [email protected]
2INSERM U768, Université René Descartes, and Hôpital Necker-Enfants Malades, Paris, France.
3Department of Microbiology, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania, USA
4Institut Universitaire d'Hématologie (IUH), Université Denis Diderot, and Hematology Laboratory AP-HP, Hôpital St-Louis, Paris, France.
5Unité de développement des lymphocytes, Département d'Immunologie, Institut Pasteur, and INSERM U668, Paris, France.
6Department of Biotherapy, Hôpital Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris (AP-HP), Université René Descartes, and INSERM, Centre d'Investigation Clinique intégré en Biothérapies, Groupe Hospitalier Universitaire Ouest, AP-HP, Paris, France.
7Hematology Laboratory and INSERM U563, Centre de Physiopathologie de Toulouse Purpan, University Hospital Purpan, Toulouse, France.
8Hematology Laboratory
9INSERM EMIU0210,
10Department of Pathology and
11Department of Pediatric Immuno-Hematology, Hôpital Necker-Enfants Malades, AP-HP, and Université René Descartes, Paris, France.
12Department of Pediatric Hematology, Oncology and Clinical Immunology, Heinrich-Heine University, Düsseldorf, Germany.
13Department of Infectious Diseases and Immunology, University Children's Hospital Munich, Munich, Germany.
14Department of Pediatrics, Louisiana State University Health Sciences Center, New Orleans, Louisiana, USA
15Department of Pediatrics, Section of Immunology, University Medical Center Utrecht, Utrecht, The Netherlands.

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10.1126/science.288.5466.669

10.1056/NEJMoa012616

10.1126/science.1070104

10.1016/S0140-6736(04)17590-9

10.1126/science.1088547

10.1126/science.1083413

Mitchell, 2004, PLoS Biol., 2, E234, 10.1371/journal.pbio.0020234

Lewinski, 2006, PLoS Pathog., 2, e60, 10.1371/journal.ppat.0020060

10.1038/nrmicro1263

10.1200/JCO.2007.12.2820

10.1053/beha.2002.0226

Kamps, 1999, Blood., 94, 1226

Asselin, 2001, Proc. Am. Soc. Clin. Oncol., 20, 367a

10.1200/JCO.2003.10.116

Printz, 1995, Gene Ther., 2, 143

10.1016/S0022-1759(02)00004-2

10.1016/S1074-7613(00)80381-7

10.1038/sj.leu.2404008

10.1038/sj.leu.2403891

10.1182/blood-2004-10-3900

10.1182/blood-2006-12-064683

Royer-Pokora, 1991, Oncogene., 6, 1887

10.1016/0092-8674(91)90383-A

10.1016/0092-8674(91)90382-9

10.1101/gad.13.20.2678

10.1038/nature01572

10.1016/S1535-6108(03)00301-5

10.1126/science.2255914

10.1126/science.1102160

Cayuela, 1996, Blood., 87, 2180, 10.1182/blood.V87.6.2180.bloodjournal8762180

Jackson, 2000, Cancer Res., 60, 2775

10.1038/ng2025

10.1200/JCO.2005.05.047

10.1038/nature05690

Dave, 2004, Science., 303, 333, 10.1126/science.1091667

10.1172/JCI31666

10.1172/JCI31659

Woods, 2006, Nature., 440, 1123, 10.1038/4401123a

Pike-Overzet, 2007, Leukemia., 21, 754, 10.1038/sj.leu.2404563

Pike-Overzet, 2006, Nature., 443, E5, 10.1038/nature05218

Thrasher, 2006, Nature., 443, E5, 10.1038/nature05219

10.1073/pnas.0603635103

10.1038/mt.2008.5

10.1038/nbt1216

10.1089/hum.2007.021

10.1038/sj.leu.2403136

10.1126/science.2814500

10.1093/nar/18.9.2793

Wang, 2008, Nucleic Acids Res., 36, e49, 10.1093/nar/gkn125

10.1101/gr.6286907

10.1016/j.ymthe.2006.03.012

10.1016/j.ymthe.2005.10.009

10.1128/JVI.79.11.6610-6619.2005

10.1016/S0092-8674(02)00864-4

Margulies, 2005, Nature., 437, 376, 10.1038/nature03959