Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: Entering a new century, do we do better? von Gennery, Andrew R., MD, Slatter, Mary A., MD, Grandin, Laure, MSc, Taupin, Pierre, MD, Cant, Andrew J., FRCP, Veys, Paul, FRCP, Amrolia, Persis J., FRCP, Gaspar, H. Bobby, PhD, Davies, E. Graham, FRCPCH, Friedrich, Wilhelm, PhD, Hoenig, Manfred, MD, Notarangelo, Luigi D., MD, Mazzolari, Evelina, MD, Porta, Fulvio, MD, Bredius, Robbert G.M., PhD, Lankester, Arjen C., PhD, Wulffraat, Nico M., PhD, Seger, Reinhard, MD, Güngör, Tayfun, MD, Fasth, Anders, PhD, Sedlacek, Petr, PhD, Neven, Benedicte, MD, Blanche, Stephane, MD, Fischer, Alain, PhD, Cavazzana-Calvo, Marina, PhD, Landais, Paul, PhD

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Haematopoietic stem cell transplantation for SCID patients: where do we stand? von Cavazzana‐Calvo, Marina, André‐Schmutz, Isabelle, Fischer, Alain

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Gene therapy for primary adaptive immune deficiencies von Fischer, Alain, MD, PhD, Hacein-Bey-Abina, Salima, PharmD, PhD, Cavazzana-Calvo, Marina, MD, PhD

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Severe combined immunodeficiency. A model disease for molecular immunology and therapy von Fischer, Alain, Le Deist, Françoise, Hacein-Bey-Abina, Salima, André-Schmutz, Isabelle, De Saint Basile, Geneviève, De Villartay, Jean-Pierre, Cavazzana-Calvo, Marina

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Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency von Hacein-Bey-Abina, Salima, Hauer, Julia, Lim, Annick, Picard, Capucine, Wang, Gary P, Berry, Charles C, Martinache, Chantal, Rieux-Laucat, Frédéric, Latour, Sylvain, Belohradsky, Bernd H, Leiva, Lily, Sorensen, Ricardo, Debré, Marianne, Casanova, Jean Laurent, Blanche, Stephane, Durandy, Anne, Bushman, Frederic D, Fischer, Alain, Cavazzana-Calvo, Marina

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Gene therapy for primary immunodeficiencies: part 1 von Cavazzana-Calvo, Marina, Fischer, Alain, Hacein-Bey-Abina, Salima, Aiuti, Alessandro

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Gene therapy for primary immunodeficiencies: Part 2 von Aiuti, Alessandro, Bacchetta, Rosa, Seger, Reinhard, Villa, Anna, Cavazzana-Calvo, Marina

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Gene therapy of inherited diseases von Fischer, Alain, Prof, Cavazzana-Calvo, Marina, Prof

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Human T-Lymphoid Progenitors Generated in a Feeder-Cell-Free Delta-Like-4 Culture System Promote T-Cell Reconstitution in NOD/SCID/γc−/− Mice von Reimann, Christian, Six, Emmanuelle, Dal-Cortivo, Liliane, Schiavo, Andrea, Appourchaux, Kevin, Lagresle-Peyrou, Chantal, de Chappedelaine, Corinne, Ternaux, Brigitte, Coulombel, Laure, Beldjord, Kheira, Cavazzana-Calvo, Marina, Andre-Schmutz, Isabelle

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Dynamics of gene-modified progenitor cells analyzed by tracking retroviral integration sites in a human SCID-X1 gene therapy trial von Wang, Gary P., Berry, Charles C., Malani, Nirav, Leboulch, Philippe, Fischer, Alain, Hacein-Bey-Abina, Salima, Cavazzana-Calvo, Marina, Bushman, Frederic D.

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Characteristics and outcome of early-onset, severe forms of Wiskott-Aldrich syndrome von Mahlaoui, Nizar, Pellier, Isabelle, Mignot, Cécile, Jais, Jean-Philippe, Bilhou-Nabéra, Chrystèle, Moshous, Despina, Neven, Bénédicte, Picard, Capucine, de Saint-Basile, Geneviève, Cavazzana-Calvo, Marina, Blanche, Stéphane, Fischer, Alain

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Sustained Correction of X-Linked Severe Combined Immunodeficiency by ex Vivo Gene Therapy von Hacein-Bey-Abina, Salima, Le Deist, Françoise, Carlier, Frédérique, Bouneaud, Cécile, Hue, Christophe, De Villartay, Jean-Pierre, Thrasher, Adrian J, Wulffraat, Nicolas, Sorensen, Ricardo, Dupuis-Girod, Sophie, Fischer, Alain, Davies, E. Graham, Kuis, Wietse, Leiva, Lilly, Cavazzana-Calvo, Marina

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Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968–99 von Antoine, Corinne, Müller, Susanna, Cant, Andrew, Cavazzana-Calvo, Marina, Veys, Paul, Vossen, Jaak, Fasth, Anders, Heilmann, Carsten, Wulffraat, Nicolas, Seger, Reinhard, Blanche, Stéphane, Friedrich, Wilhelm, Abinun, Mario, Davies, Graham, Bredius, Robert, Schulz, Ansgar, Landais, Paul, Fischer, Alain

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Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy von Deichmann, Annette, Hacein-Bey-Abina, Salima, Schmidt, Manfred, Garrigue, Alexandrine, Brugman, Martijn H, Hu, Jingqiong, Glimm, Hanno, Gyapay, Gabor, Prum, Bernard, Fraser, Christopher C, Fischer, Nicolas, Schwarzwaelder, Kerstin, Siegler, Maria-Luise, de Ridder, Dick, Pike-Overzet, Karin, Howe, Steven J, Thrasher, Adrian J, Wagemaker, Gerard, Abel, Ulrich, Staal, Frank J T, Delabesse, Eric, Villeval, Jean-Luc, Aronow, Bruce, Hue, Christophe, Prinz, Claudia, Wissler, Manuela, Klanke, Chuck, Weissenbach, Jean, Alexander, Ian, Fischer, Alain, von Kalle, Christof, Cavazzana-Calvo, Marina

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Artemis, a Novel DNA Double-Strand Break Repair/V(D)J Recombination Protein, Is Mutated in Human Severe Combined Immune Deficiency von Moshous, Despina, Callebaut, Isabelle, de Chasseval, Régina, Corneo, Barbara, Cavazzana-Calvo, Marina, Le Deist, Françoise, Tezcan, Ilhan, Sanal, Ozden, Bertrand, Yves, Philippe, Noel, Fischer, Alain, de Villartay, Jean-Pierre

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Gene Therapy of Human Severe Combined Immunodeficiency (SCID)-X1 Disease von Cavazzana-Calvo, Marina, Hacein-Bey, Salima, Geneviève de Saint Basile, Gross, Fabian, Yvon, Eric, Nusbaum, Patrick, Selz, Françoise, Hue, Christophe, Certain, Stéphanie, Casanova, Jean-Laurent, Bousso, Philippe, Le Deist, Françoise, Fischer, Alain

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A method to sequence and quantify DNA integration for monitoring outcome in gene therapy von Brady, Troy, Roth, Shoshannah L., Malani, Nirav, Wang, Gary P., Berry, Charles C., Leboulch, Philippe, Hacein-Bey-Abina, Salima, Cavazzana-Calvo, Marina, Papapetrou, Eirini P., Sadelain, Michel, Savilahti, Harri, Bushman, Frederic D.

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Primary Microcephaly, Impaired DNA Replication, and Genomic Instability Caused by Compound Heterozygous ATR Mutations von Mokrani-Benhelli, Houda, Gaillard, Laetitia, Biasutto, Patricia, Le Guen, Tangui, Touzot, Fabien, Vasquez, Nadia, Komatsu, Jun, Conseiller, Emmanuel, Pïcard, Capucine, Gluckman, Eliane, Francannet, Christine, Fischer, Alain, Durandy, Anne, Soulier, Jean, de Villartay, Jean-Pierre, Cavazzana-Calvo, Marina, Revy, Patrick

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Hematopoietic Stem Cell Transplantation in Hemophagocytic Lymphohistiocytosis: A Single-Center Report of 48 Patients von Ouachee-Chardin, Marie, Elie, Caroline, de Saint Basile, Genevieve, Le Deist, Francoise, Mahlaoui, Nizar, Picard, Capucine, Neven, Benedicte, Casanova, Jean-Laurent, Tardieu, Marc, Cavazzana-Calvo, Marina, Blanche, Stephane, Fischer, Alain

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Long-term outcome following hematopoietic stem-cell transplantation in Wiskott-Aldrich syndrome: collaborative study of the European Society for Immunodeficiencies and European Gro... von Ozsahin, Hulya, Cavazzana-Calvo, Marina, Notarangelo, Luigi D., Schulz, Ansgar, Thrasher, Adrian J., Mazzolari, Evelina, Slatter, Mary A., Le Deist, Francoise, Blanche, Stephane, Veys, Paul, Fasth, Anders, Bredius, Robbert, Sedlacek, Petr, Wulffraat, Nico, Ortega, Juan, Heilmann, Carsten, O’Meara, Anne, Wachowiak, Jacek, Kalwak, Krzysztof, Matthes-Martin, Susanne, Gungor, Tayfun, Ikinciogullari, Aydan, Landais, Paul, Cant, Andrew J., Friedrich, Wilhelm, Fischer, Alain

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