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Outcomes Following Gene Therapy in Patients With Severe Wiskott-Aldrich Syndrome

Hacein-Bey Abina, Salima, Gaspar, H. Bobby, Blondeau, Johanna, Caccavelli, Laure, Charrier, Sabine, Buckland, Karen, Picard, Capucine, Six, Emmanuelle, Himoudi, Nourredine, Gilmour, Kimberly, McNicol, Anne-Marie, Hara, Havinder, Xu-Bayford, Jinhua, Rivat, Christine, Touzot, Fabien, Mavilio, Fulvio, Lim, Annick, Treluyer, Jean-Marc, Héritier, Sébastien, Lefrère, Francois, Magalon, Jeremy, Pengue-Koyi, Isabelle, Honnet, Géraldine, Blanche, Stéphane, Sherman, Eric A., Male, Frances, Berry, Charles, Malani, Nirav, Bushman, Frederic D., Fischer, Alain, Thrasher, Adrian J., Galy, Anne, Cavazzana, Marina·2015·JAMA
DOI 10.1001/jama.2015.3253PMC PMC4942841
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Research works printed but not linked: 39

  1. [1]Albert MH Notarangelo LD Ochs HD Clinical spectrum, pathophysiology and treatment of the Wiskott-Aldrich syndrome Current opinion in hematology 1 2011 18 1 42 48 21076297
  2. [2]Thrasher AJ Burns SO WASP: a key immunological multitasker Nat Rev Immunol 3 2010 10 3 182 192 20182458
  3. [3]Zhu Q Zhang M Blaese RM The Wiskott-Aldrich syndrome and X-linked congenital thrombocytopenia are caused by mutations of the same gene Blood 11 15 1995 86 10 3797 3804 7579347
  4. [4]Imai K Morio T Zhu Y Clinical course of patients with WASP gene mutations Blood 1 15 2004 103 2 456 464 12969986
  5. [5]Mahlaoui N Pellier I Mignot C Characteristics and outcome of early-onset, severe forms of Wiskott-Aldrich syndrome Blood 2 28 2013 121 9 1510 1516 23264593
  6. [6]Pai SY Notarangelo LD Hematopoietic cell transplantation for Wiskott-Aldrich syndrome: advances in biology and future directions for treatment Immunol Allergy Clin North Am 5 2010 30 2 179 194 20493395 PMC2930258
  7. [7]Kobayashi R Ariga T Nonoyama S Outcome in patients with Wiskott-Aldrich syndrome following stem cell transplantation: an analysis of 57 patients in Japan Br J Haematol 11 2006 135 3 362 366 17032176
  8. [8]Friedrich W Schutz C Schulz A Benninghoff U Honig M Results and long-term outcome in 39 patients with Wiskott-Aldrich syndrome transplanted from HLA-matched and -mismatched donors Immunol Res 2009 44 1–3 18 24 18846321
  9. [9]Gennery AR Slatter MA Grandin L Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: entering a new century, do we do better? J Allergy Clin Immunol 9 2010 126 3 602 610 e601 611 20673987
  10. [10]Moratto D Giliani S Bonfim C Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980–2009: an international collaborative study Blood 8 11 2011 118 6 1675 1684 21659547 PMC3156052
  11. [12]Charrier S Dupre L Scaramuzza S Lentiviral vectors targeting WASp expression to hematopoietic cells, efficiently transduce and correct cells from WAS patients Gene Ther 3 2007 14 5 415 428 17051251
  12. [13]Marangoni F Bosticardo M Charrier S Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models Mol Ther 6 2009 17 6 1073 1082 19259069 PMC2835187
  13. [14]Zanta-Boussif MA Charrier S Brice-Ouzet A Validation of a mutated PRE sequence allowing high and sustained transgene expression while abrogating WHV-X protein synthesis: application to the gene therapy of WAS Gene Ther 5 2009 16 5 605 619 19262615
  14. [15]Aiuti A Biasco L Scaramuzza S Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome Science 8 23 2013 341 6148 1233151 23845947 PMC4375961
  15. [16]Merten OW Charrier S Laroudie N Large-scale manufacture and characterization of a lentiviral vector produced for clinical ex vivo gene therapy application Hum Gene Ther 3 2011 22 3 343 356 21043787
  16. [17]Cavazzana-Calvo M Hacein-Bey S de Saint Basile G Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease Science 4 28 2000 288 5466 669 672 10784449
  17. [19]Lim A Baron V Ferradini L Bonneville M Kourilsky P Pannetier C Combination of MHC-peptide multimer-based T cell sorting with the Immunoscope permits sensitive ex vivo quantitation and follow-up of human CD8+ T cell immune responses J Immunol Methods 3 1 2002 261 1–2 177 194 11861076
  18. [20]Hacein-Bey-Abina S Le Deist F Carlier F Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy The New England journal of medicine 4 18 2002 346 16 1185 1193 11961146
  19. [21]Poulin JF Sylvestre M Champagne P Evidence for adequate thymic function but impaired naive T-cell survival following allogeneic hematopoietic stem cell transplantation in the absence of chronic graft-versus-host disease Blood 12 15 2003 102 13 4600 4607 12933579
  20. [22]Charrier S Ferrand M Zerbato M Quantification of lentiviral vector copy numbers in individual hematopoietic colony-forming cells shows vector dose-dependent effects on the frequency and level of transduction Gene Ther 5 2011 18 5 479 487 21160533 PMC3130191
  21. [23]Wang GP Ciuffi A Leipzig J Berry CC Bushman FD HIV integration site selection: analysis by massively parallel pyrosequencing reveals association with epigenetic modifications Genome Res 8 2007 17 8 1186 1194 17545577 PMC1933515
  22. [24]Wang GP Garrigue A Ciuffi A DNA bar coding and pyrosequencing to analyze adverse events in therapeutic gene transfer Nucleic Acids Res 5 2008 36 9 e49 18411205 PMC2396413
  23. [25]Berry CC Gillet NA Melamed A Gormley N Bangham CR Bushman FD Estimating abundances of retroviral insertion sites from DNA fragment length data Bioinformatics 3 15 2012 28 6 755 762 22238265 PMC3307109
  24. [26]Berry C Hannenhalli S Leipzig J Bushman FD Selection of target sites for mobile DNA integration in the human genome PLoS Comput Biol 11 24 2006 2 11 e157 17166054 PMC1664696
  25. [27]Berry CC Ocwieja KE Malani N Bushman FD Comparing DNA integration site clusters with scan statistics Bioinformatics 6 1 2014 30 11 1493 1500 24489369 PMC4029028
  26. [28]Rengan R Ochs HD Sweet LI Actin cytoskeletal function is spared, but apoptosis is increased, in WAS patient hematopoietic cells Blood 2 15 2000 95 4 1283 1292 10666201
  27. [29]Westerberg LS de la Fuente MA Wermeling F WASP confers selective advantage for specific hematopoietic cell populations and serves a unique role in marginal zone B-cell homeostasis and function Blood 11 15 2008 112 10 4139 4147 18772454 PMC2581999
  28. [30]Yamaguchi K Ariga T Yamada M Mixed chimera status of 12 patients with Wiskott-Aldrich syndrome (WAS) after hematopoietic stem cell transplantation: evaluation by flow cytometric analysis of intracellular WAS protein expression Blood 8 15 2002 100 4 1208 1214 12149199
  29. [31]Maillard MH Cotta-de-Almeida V Takeshima F The Wiskott-Aldrich syndrome protein is required for the function of CD4(+)CD25(+)Foxp3(+) regulatory T cells J Exp Med 2 19 2007 204 2 381 391 17296786 PMC2118715
  30. [32]Marangoni F Trifari S Scaramuzza S WASP regulates suppressor activity of human and murine CD4(+)CD25(+)FOXP3(+) natural regulatory T cells J Exp Med 2 19 2007 204 2 369 380 17296785 PMC2118740
  31. [33]Molina IJ Sancho J Terhorst C Rosen FS Remold-O’Donnell E T cells of patients with the Wiskott-Aldrich syndrome have a restricted defect in proliferative responses J Immunol 10 15 1993 151 8 4383 4390 8409409
  32. [34]Snapper SB Rosen FS Mizoguchi E Wiskott-Aldrich syndrome protein-deficient mice reveal a role for WASP in T but not B cell activation Immunity Jul 1998 9 1 81 91 9697838
  33. [35]Zhang J Shehabeldin A da Cruz LA Antigen receptor-induced activation and cytoskeletal rearrangement are impaired in Wiskott-Aldrich syndrome protein-deficient lymphocytes J Exp Med 11 1 1999 190 9 1329 1342 10544204 PMC2195687
  34. [36]Burns S Thrasher AJ Blundell MP Machesky L Jones GE Configuration of human dendritic cell cytoskeleton by Rho GTPases, the WAS protein, and differentiation Blood 8 15 2001 98 4 1142 1149 11493463
  35. [37]Calle Y Chou HC Thrasher AJ Jones GE Wiskott-Aldrich syndrome protein and the cytoskeletal dynamics of dendritic cells J Pathol 11 2004 204 4 460 469 15495215
  36. [38]Duchamp M Sterlin D Diabate A B-cell subpopulations in children: National reference values Immunity, Inflammation and Disease 2014 2 3 131 140 PMC4257758 25505547
  37. [39]Schroder AR Shinn P Chen H Berry C Ecker JR Bushman F HIV-1 integration in the human genome favors active genes and local hotspots Cell 8 23 2002 110 4 521 529 12202041
  38. [40]Hacein-Bey-Abina S Hauer J Lim A Efficacy of gene therapy for X-linked severe combined immunodeficiency The New England journal of medicine 7 22 2010 363 4 355 364 20660403 PMC2957288
  39. [42]Ozsahin H Cavazzana-Calvo M Notarangelo LD Long-term outcome following hematopoietic stem-cell transplantation in Wiskott-Aldrich syndrome: collaborative study of the European Society for Immunodeficiencies and European Group for Blood and Marrow Transplantation Blood 1 1 2008 111 1 439 445 17901250

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Metadata

Authors
Hacein-Bey Abina, S., Gaspar, H. B., Blondeau, J., Caccavelli, L., Charrier, S., Buckland, K., Picard, C., Six, E., Himoudi, N., Gilmour, K., McNicol, A.-M., Hara, H., Xu-Bayford, J., Rivat, C., Touzot, F., Mavilio, F., Lim, A., Treluyer, J.-M., Héritier, S., Lefrère, F., Magalon, J., Pengue-Koyi, I., Honnet, G., Blanche, S., Sherman, E. A., Male, F., Berry, C., Malani, N., Bushman, F. D., Fischer, A., Thrasher, A. J., Galy, A., & Cavazzana, M.
Affiliations
  • Biotherapy Department, Necker Children’s Hospital, Assistance Publique–Hôpitaux de Paris, Paris, France2Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, Assistance Publique–Hôpitaux de Paris, INSERM, Paris, France3Unité de
  • Section of Molecular and Cellular Immunology, University College London Institute of Child Health, London, England6Department of Clinical Immunology, Hospital National Health Service Foundation Trust, London, England
  • Biotherapy Department, Necker Children’s Hospital, Assistance Publique–Hôpitaux de Paris, Paris, France2Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, Assistance Publique–Hôpitaux de Paris, INSERM, Paris, France
  • INSERM U951, Unité Mixte de Recherche S951, Molecular Immunology and Innovative Biotherapies, University of Evry, Evry, France8Genethon, Evry, France
  • Centre d’Étude des Déficits Immunitaires, Hôpital Necker-Enfants Malades, Assistance Publique–Hôpitaux de Paris, Paris, France10Paris Descartes–Sorbonne Paris Cité University, Imagine Institute, Paris, France11Immunology and Pediatric Hematology Departmen
  • Paris Descartes–Sorbonne Paris Cité University, Imagine Institute, Paris, France12INSERM Unité Mixte de Recherche 1163, Laboratory of Human Lymphohematopoiesis, Paris, France
  • Department of Clinical Immunology, Hospital National Health Service Foundation Trust, London, England
  • Biotherapy Department, Necker Children’s Hospital, Assistance Publique–Hôpitaux de Paris, Paris, France2Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, Assistance Publique–Hôpitaux de Paris, INSERM, Paris, France10Paris D
  • Genethon, Evry, France
  • Groupe Immunoscope, Immunology Department, Institut Pasteur, Paris, France
  • Clinical Research Center Necker–Enfants Malades and Cochin Hospital Assistance Publique–Hôpitaux de Paris, Paris Descartes University
  • Paris Descartes–Sorbonne Paris Cité University, Imagine Institute, Paris, France11Immunology and Pediatric Hematology Department, Assistance Publique–Hôpitaux de Paris, Paris, France
  • Biotherapy Department, Necker Children’s Hospital, Assistance Publique–Hôpitaux de Paris, Paris, France
  • Department of Microbiology, University of Pennsylvania School of Medicine, Philadelphia
  • Paris Descartes–Sorbonne Paris Cité University, Imagine Institute, Paris, France11Immunology and Pediatric Hematology Department, Assistance Publique–Hôpitaux de Paris, Paris, France12INSERM Unité Mixte de Recherche 1163, Laboratory of Human Lymphohematop
Venue
JAMA
Publisher
American Medical Association (AMA)
Published
2015-04-21
Volume
313
Issue
15
Pages
1550
ISSN
0098-7484
DOI
10.1001/jama.2015.3253
PMC
PMC4942841
Cited by
357
References
3
Language
en

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