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Recombination-activating gene 1 (Rag1)-deficient mice with severe combined immunodeficiency treated with lentiviral gene therapy demonstrate autoimmune Omenn-like syndrome.
van Til, Niek P; Sarwari, Roya; Visser, Trudi P; Hauer, Julia; Lagresle-Peyrou, Chantal; van der Velden, Guus; Malshetty, Vidyasagar; Cortes, Patricia; Jollet, Arnaud; Danos, Olivier; Cassani, Barbara; Zhang, Fang; Thrasher, Adrian J; Fontana, Elena; Poliani, Pietro L; Cavazzana, Marina; Verstegen, Monique M A; Villa, Anna; Wagemaker, Gerard.
Afiliação
  • van Til NP; Department of Hematology, Erasmus University Medical Center, Rotterdam, The Netherlands.
  • Sarwari R; Department of Hematology, Erasmus University Medical Center, Rotterdam, The Netherlands.
  • Visser TP; Department of Hematology, Erasmus University Medical Center, Rotterdam, The Netherlands.
  • Hauer J; INSERM U768, Université René Descartes, and Hôpital Necker-Enfants Malades, Paris, France; Department of Pediatric Oncology, Hematology and Clinical Immunology, Center for Child and Adolescent Health, Heinrich Heine University, Düsseldorf, Germany.
  • Lagresle-Peyrou C; INSERM U768, Université René Descartes, and Hôpital Necker-Enfants Malades, Paris, France.
  • van der Velden G; Department of Hematology, Erasmus University Medical Center, Rotterdam, The Netherlands.
  • Malshetty V; Immunology Institute, Department of Medicine, Mount Sinai School of Medicine, New York, NY.
  • Cortes P; Immunology Institute, Department of Medicine, Mount Sinai School of Medicine, New York, NY.
  • Jollet A; INSERM U781 Laboratoire de transfert de gènes, Hôpital Necker-Enfants Malades, Paris, France.
  • Danos O; INSERM U781 Laboratoire de transfert de gènes, Hôpital Necker-Enfants Malades, Paris, France.
  • Cassani B; CNR-IRGB, Milan Unit, Milan, Italy; Humanitas Clinical and Research Center, Milan, Italy.
  • Zhang F; Molecular Immunology Unit, Centre for Immunodeficiency, Institute of Child Health, University College London, London, United Kingdom.
  • Thrasher AJ; Molecular Immunology Unit, Centre for Immunodeficiency, Institute of Child Health, University College London, London, United Kingdom.
  • Fontana E; Department of Pathology, University of Brescia, Brescia, Italy.
  • Poliani PL; Department of Pathology, University of Brescia, Brescia, Italy.
  • Cavazzana M; INSERM U768, Université René Descartes, and Hôpital Necker-Enfants Malades, Paris, France; Department 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 H
  • Verstegen MM; Department of Hematology, Erasmus University Medical Center, Rotterdam, The Netherlands.
  • Villa A; CNR-IRGB, Milan Unit, Milan, Italy; Telethon Institute for Gene Therapy-H San Raffaele, Milan, Italy.
  • Wagemaker G; Department of Hematology, Erasmus University Medical Center, Rotterdam, The Netherlands. Electronic address: g.wagemaker@genetherapy.nl.
J Allergy Clin Immunol ; 133(4): 1116-23, 2014 Apr.
Article em En | MEDLINE | ID: mdl-24332219
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Terapia Genética / Imunodeficiência Combinada Severa / Lentivirus / Proteínas de Homeodomínio / Vetores Genéticos Tipo de estudo: Prognostic_studies Limite: Animals / Female / Humans / Male Idioma: En Ano de publicação: 2014 Tipo de documento: Article
Texto completo
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Terapia Genética / Imunodeficiência Combinada Severa / Lentivirus / Proteínas de Homeodomínio / Vetores Genéticos Tipo de estudo: Prognostic_studies Limite: Animals / Female / Humans / Male Idioma: En Ano de publicação: 2014 Tipo de documento: Article