Stable and Functional Lymphoid Reconstitution in Artemis-deficient Mice Following Lentiviral Artemis Gene Transfer Into Hematopoietic Stem Cells

Molecular Therapy - Tập 16 - Trang 1490-1499 - 2008
Fatine Benjelloun1, Alexandrine Garrigue1, Corinne Demerens-de Chappedelaine1, Pauline Soulas-Sprauel1, Michele Malassis-Séris1, Daniel Stockholm2, Julia Hauer1, Johanna Blondeau2, Julie Rivière1, Annick Lim3, Marc Le Lorc'h4, Serge Romana4, Nicole Brousse5, Frederique Pâques6, Anne Galy2, Pierre Charneau7, Alain Fischer1,8, Jean-Pierre de Villartay1,9, Marina Cavazzana-Calvo1,10
1Institut National de la Santé et de la Recherche (INSERM), U768, Université Paris Descartes, Hôpital Necker-Enfants Malades, Paris,France
2Généthon, Evry, France
3Unité de Développement des Lymphocytes, Département d'Immunologie, INSERM, U668, Institut Pasteur, Paris, France
4Service d'Histologie-Embryologie et Cytogénétique Assistance Publique, Hôpitaux de Paris (AP/HP), Hôpital Necker-Enfants Malades, Paris, France
5Service d'Anatomie et Cytologie Pathologique, AP/HP, Hôpital Necker-Enfants Malades, Paris, France
6Cellectis SA, Romainville, France
7Département de Virologie Moléculaire et Vectorologie, INSERM, U668, Institut Pasteur, Paris, France
8Unité d'Immunologie et Hématologie Pédiatrique, AP/HP, Hôpital Necker-Enfants Malades, Paris, France
9Service d'Hématologie, AP/HP, Hôpital Necker-Enfants Malades, Paris, France
10Département de Biotherapie, AP/HP, Hôpital Necker-Enfants Malades, Paris, France

Tài liệu tham khảo

Fischer, 2000, Severe combined immunodeficiencies (SCID), Clin Exp Immunol, 122, 143, 10.1046/j.1365-2249.2000.01359.x Schwarz, 1996, RAG mutations in human B cell-negative SCID, Science, 274, 97, 10.1126/science.274.5284.97 Moshous, 2001, Artemis, a novel DNA double-strand break repair/V(D)J recombination protein, is mutated in human severe combined immune deficiency, Cell, 105, 177, 10.1016/S0092-8674(01)00309-9 Revy, 2005, The repair of DNA damages/modifications during the maturation of the immune system: lessons from human primary immunodeficiency disorders and animal models, Adv Immunol, 87, 237, 10.1016/S0065-2776(05)87007-5 Nicolas, 1996, Lack of detectable defect in DNA double-strand break repair and DNA-dependent protein kinase activity in radiosensitive human severe combined immunodeficiency fibroblasts, Eur J Immunol, 26, 1118, 10.1002/eji.1830260524 Kobayashi, 2003, Novel Artemis gene mutations of radiosensitive severe combined immunodeficiency in Japanese families, Hum Genet, 112, 348, 10.1007/s00439-002-0897-x Noordzij, 2003, Radiosensitive SCID patients with Artemis gene mutations show a complete B-cell differentiation arrest at the pre-B-cell receptor checkpoint in bone marrow, Blood, 101, 1446, 10.1182/blood-2002-01-0187 Moshous, 2003, Partial T and B lymphocyte immunodeficiency and predisposition to lymphoma in patients with hypomorphic mutations in Artemis, J Clin Invest, 111, 381, 10.1172/JCI16774 Antoine, 2003, Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968–99, Lancet, 361, 553, 10.1016/S0140-6736(03)12513-5 Rooney, 2002, Leaky Scid phenotype associated with defective V(D)J coding end processing in Artemis-deficient mice, Mol Cell, 10, 1379, 10.1016/S1097-2765(02)00755-4 Li, 2005, Targeted disruption of the Artemis murine counterpart results in SCID and defective V(D)J recombination that is partially corrected with bone marrow transplantation, J Immunol, 174, 2420, 10.4049/jimmunol.174.4.2420 Rooney, 2003, Defective DNA repair and increased genomic instability in Artemis-deficient murine cells, J Exp Med, 197, 553, 10.1084/jem.20021891 Musio, 2005, Damaging-agent sensitivity of Artemis-deficient cell lines, Eur J Immunol, 35, 1250, 10.1002/eji.200425555 Darroudi, 2007, Role of Artemis in DSB repair and guarding chromosomal stability following exposure to ionizing radiation at different stages of cell cycle, Mutat Res, 615, 111, 10.1016/j.mrfmmm.2006.11.029 Sirven, 2001, Enhanced transgene expression in cord blood CD34(+)-derived hematopoietic cells, including developing T cells and NOD/SCID mouse repopulating cells, following transduction with modified trip lentiviral vectors, Mol Ther, 3, 438, 10.1006/mthe.2001.0282 Li, 2002, Murine leukemia induced by retroviral gene marking, Science, 296, 497, 10.1126/science.1068893 Hacein-Bey-Abina, 2003, LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1, Science, 302, 415, 10.1126/science.1088547 Wu, 2003, Transcription start regions in the human genome are favored targets for MLV integration, Science, 300, 1749, 10.1126/science.1083413 Calmels, 2005, Recurrent retroviral vector integration at the Mds1/Evi1 locus in nonhuman primate hematopoietic cells, Blood, 106, 2530, 10.1182/blood-2005-03-1115 Cavazzana-Calvo, 2007, Gene therapy for severe combined immunodeficiency: are we there yet?, J Clin Invest, 117, 1456, 10.1172/JCI30953 Morre, 1997, NADH oxidase activity from sera altered by capsaicin is widely distributed among cancer patients, Arch Biochem Biophys, 342, 224, 10.1006/abbi.1997.0110 Wang, 2003, Sera from cancer patients contain two oscillating ECTO-NOX activities with different period lengths, Cancer Lett, 190, 135, 10.1016/S0304-3835(02)00616-X Lagresle-Peyrou, 2006, Long-term immune reconstitution in RAG-1-deficient mice treated by retroviral gene therapy: a balance between efficiency and toxicity, Blood, 107, 63, 10.1182/blood-2005-05-2032 Mostoslavsky, 2006, Complete correction of murine Artemis immunodeficiency by lentiviral vector-mediated gene transfer, Proc Natl Acad Sci USA, 103, 16406, 10.1073/pnas.0608130103 Will, 2007, Importance of murine study design for testing toxicity of retroviral vectors in support of phase I trials, Mol Ther, 15, 782, 10.1038/sj.mt.6300083 Siapati, 2007, Murine leukemia following irradiation conditioning for transplantation of lentivirally-modified hematopoietic stem cells, Eur J Haematol, 78, 303, 10.1111/j.1600-0609.2006.00813.x Rooney, 2004, Artemis and p53 cooperate to suppress oncogenic N-myc amplification in progenitor B cells, Proc Natl Acad Sci USA, 101, 2410, 10.1073/pnas.0308757101 Lagresle-Peyrou, 2007, Lentiviral gene-corrected CD34+ cells isolated from Artemis-deficient patients differentiate into functional B cells following injection into NOD-SCID mice, Mol Ther, 16, 396, 10.1038/sj.mt.6300353 Zennou, 2000, HIV-1 genome nuclear import is mediated by a central DNA flap, Cell, 101, 173, 10.1016/S0092-8674(00)80828-4 Pannetier, 1993, The sizes of the CDR3 hypervariable regions of the murine T-cell receptor beta chains vary as a function of the recombined germ-line segments, Proc Natl Acad Sci USA, 90, 4319, 10.1073/pnas.90.9.4319 Charrier, 2005, A lentiviral vector encoding the human Wiskott-Aldrich syndrome protein corrects immune and cytoskeletal defects in WASP knockout mice, Gene Ther, 12, 597, 10.1038/sj.gt.3302440 Schmidt, 2002, Polyclonal long-term repopulating stem cell clones in a primate model, Blood, 100, 2737, 10.1182/blood-2002-02-0407 Padilla-Nash, 2006, Spectral karyotyping analysis of human and mouse chromosomes, Nat Protoc, 1, 3129, 10.1038/nprot.2006.358