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ArticlesPeer ReviewedFull Text
Gene Therapy in a Patient with Sickle Cell Disease
Author / Creator:Ribeil, Jean-Antoine ; Hacein-Bey-Abina, Salima ; Payen, Emmanuel ; Magnani, Alessandra ; Semeraro, Michaela ; Magrin, Elisa ; Caccavelli, Laure ; Neven, Benedicte ; Bourget, Philippe ; El Nemer, Wassim ; Bartolucci, Pablo ; Weber, Leslie ; Puy, Hervé ; Meritet, Jean-François ; Grevent, David ; Beuzard, Yves ; Chrétien, Stany ; Lefebvre, Thibaud ; Ross, Robert W ; Negre, Olivier ; Veres, Gabor ; Sandler, Laura ; Soni, Sandeep ; de Montalembert, Mariane ; Blanche, Stéphane ; Leboulch, Philippe ; Cavazzana, Marina
Publisher:United States: Massachusetts Medical Society
Journal title:The New England journal of medicine, 2017-03, Vol.376 (9), p.848-855
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Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial
Author / Creator:Magrin, Elisa ; Semeraro, Michaela ; Hebert, Nicolas ; Joseph, Laure ; Magnani, Alessandra ; Chalumeau, Anne ; Gabrion, Aurélie ; Roudaut, Cécile ; Marouene, Jouda ; Lefrere, Francois ; Diana, Jean-Sebastien ; Denis, Adeline ; Neven, Bénédicte ; Funck-Brentano, Isabelle ; Negre, Olivier ; Renolleau, Sylvain ; Brousse, Valentine ; Kiger, Laurent ; Touzot, Fabien ; Poirot, Catherine ; Bourget, Philippe ; El Nemer, Wassim ; Blanche, Stéphane ; Tréluyer, Jean-Marc ; Asmal, Mohammed ; Walls, Courtney ; Beuzard, Yves ; Schmidt, Manfred ; Hacein-Bey-Abina, Salima ; Asnafi, Vahid ; Guichard, Isabelle ; Poirée, Maryline ; Monpoux, Fabrice ; Touraine, Philippe ; Brouzes, Chantal ; de Montalembert, Mariane ; Payen, Emmanuel ; Six, Emmanuelle ; Ribeil, Jean-Antoine ; Miccio, Annarita ; Bartolucci, Pablo ; Leboulch, Philippe ; Cavazzana, Marina
Publisher:New York: Nature Publishing Group US
Journal title:Nature medicine, 2022-01, Vol.28 (1), p.81-88
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Correction to: Ex vivo generated human T-lymphoid progenitors as a tool to accelerate immune reconstitution after partially HLA compatible hematopoietic stem cell transplantation or after gene therapy
Author / Creator:André, Isabelle ; Simons, Laura ; Ma, Kuiying ; Moirangthem, Ranjita Devi ; Diana, Jean-Sébastien ; Magrin, Elisa ; Couzin, Chloé ; Magnani, Alessandra ; Lagresle-Peyrou, Chantal ; Cavazzana, Marina
Publisher:London: Nature Publishing Group UK
Journal title:Bone marrow transplantation (Basingstoke), 2022-05, Vol.57 (5), p.851-851
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Severe inflammation and lineage skewing are associated with poor engraftment of engineered hematopoietic stem cells in patients with sickle cell disease
Author / Creator:Sobrino, Steicy ; Joseph, Laure ; Magrin, Elisa ; Chalumeau, Anne ; Hebert, Nicolas ; Corsia, Alice ; Denis, Adeline ; Roudaut, Cécile ; Aussel, Clotilde ; Leblanc, Olivia ; Brusson, Mégane ; Felix, Tristan ; Diana, Jean-Sebastien ; Petrichenko, Angelina ; El Etri, Jana ; Godard, Auria ; Tibi, Eden ; Manceau, Sandra ; Treluyer, Jean Marc ; Mavilio, Fulvio ; Bushman, Frederic D. ; Marcais, Ambroise ; Castelle, Martin ; Neven, Benedicte ; Hermine, Olivier ; Renolleau, Sylvain ; Magnani, Alessandra ; Asnafi, Vahid ; El Nemer, Wassim ; Bartolucci, Pablo ; Six, Emmanuelle ; Semeraro, Michaela ; Miccio, Annarita ; Cavazzana, Marina
Publisher:London: Nature Publishing Group UK
Journal title:Nature communications, 2025-04, Vol.16 (1), p.3137-17, Article 3137
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Ex vivo generated human T-lymphoid progenitors as a tool to accelerate immune reconstitution after partially HLA compatible hematopoietic stem cell transplantation or after gene therapy
Author / Creator:André, Isabelle ; Simons, Laura ; Ma, Kuiying ; Moirangthem, Ranjita Devi ; Diana, Jean-Sébastien ; Magrin, Elisa ; Couzin, Chloé ; Magnani, Alessandra ; Cavazzana, Marina
Publisher:London: Nature Publishing Group UK
Journal title:Bone marrow transplantation (Basingstoke), 2019-08, Vol.54 (Suppl 2), p.749-755
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559. Induction of Fetal Hemoglobin in Adult Erythroblasts by Genome Editing of the Beta-Globin Locus
Author / Creator:Antoniani, Chiara ; Lattanzi, Annalisa ; Pavani, Giulia ; Meneghini, Vasco ; Magrin, Elisa ; Weber, Leslie ; Porteus, Matthew ; Cavazzana, Marina ; Mavilio, Fulvio ; Miccio, Annarita
Publisher:Elsevier Inc
Journal title:Molecular therapy, 2016-05, Vol.24, p.S223-S224
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Gene Therapy in Patients with Transfusion-Dependent [beta]-Thalassemia
Author / Creator:Thompson, Alexis A ; Walters, Mark C ; Kwiatkowski, Janet ; Rasko, John EJ ; Ribeil, Jean-Antoine ; Hongeng, Suradej ; Magrin, Elisa ; Schiller, Gary J ; Payen, Emmanuel ; Semeraro, Michaela ; Moshous, Despina ; Lefrere, Francois ; Puy, Hervé ; Bourget, Philippe ; Magnani, Alessandra ; Caccavelli, Laure ; Diana, Jean-Sébastien ; Suarez, Felipe ; Monpoux, Fabrice ; Brousse, Valentine ; Poirot, Catherine ; Brouzes, Chantal ; Meritet, Jean-François ; Pondarré, Corinne ; Beuzard, Yves ; Chrétien, Stany ; Lefebvre, Thibaud ; Teachey, David T ; Anurathapan, Usanarat ; Ho, P Joy ; von Kalle, Christof ; Kletzel, Morris ; Vichinsky, Elliott ; Soni, Sandeep ; Veres, Gabor ; Negre, Olivier ; Ross, Robert W ; Davidson, David ; Petrusich, Alexandria ; Sandler, Laura ; Asmal, Mohammed ; Hermine, Olivier ; De Montalembert, Mariane ; Hacein-Bey-Abina, Salima ; Blanche, Stéphane ; Leboulch, Philippe ; Cavazzana, Marina
Publisher:Boston: Massachusetts Medical Society
Journal title:The New England journal of medicine, 2018-04, Vol.378 (16), p.1479
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231. Mixed Chimerism After Allogeneic Hematopoietic Stem Cell Transplantation in Sickle Cell Disease: Preliminary Results on Peripheral Blood Sorted Subpopulations and Erythroid Progenitors
Author / Creator:Magnani, Alessandra ; Caccavelli, Laure ; Fusaro, Mathieu ; Magalon, Jeremy ; Magrin, Elisa ; Six, Emmanuelle ; Touzot, Fabien ; Couzin, Chloé ; Ribeil, Jean Antoine ; Arnaud, Cécile ; Pondarré, Corinne ; Kamdem, Annie ; Bernaudin, Françoise ; Vernant, Jean Paul ; Gluckman, Eliane ; Bories, Dominique ; Cavazzana, Marina
Publisher:Elsevier Inc
Journal title:Molecular therapy, 2016-05, Vol.24, p.S90-S91
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754. Exploring the Human Hematopoietic Hierarchy Through Retroviral Integration Sites Tracking in the Wiskott Aldrich Syndrome Gene Therapy Trial
Author / Creator:Six, Emmanuelle ; Caccavelli, Laure ; Roudaut, Cecile ; Male, Frances ; Wu, Yinghua ; Cagnard, Nicolas ; Magnani, Alessandra ; Touzot, Fabien ; Magrin, Elisa ; Galy, Anne ; André-Schmutz, Isabelle ; Abina, Salima Hacein-Bey ; Jais, Jean-Philippe ; Berry, Charles C. ; Bushman, Frederic D. ; Cavazzana, Marina
Publisher:Elsevier Inc
Journal title:Molecular therapy, 2016-05, Vol.24, p.S298-S298
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An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype
Author / Creator:Weber, Leslie ; Poletti, Valentina ; Magrin, Elisa ; Antoniani, Chiara ; Martin, Samia ; Bayard, Charles ; Sadek, Hanem ; Felix, Tristan ; Meneghini, Vasco ; Antoniou, Michael N. ; El-Nemer, Wassim ; Mavilio, Fulvio ; Cavazzana, Marina ; Andre-Schmutz, Isabelle ; Miccio, Annarita
Publisher:United States: Elsevier Inc
Journal title:Molecular therapy. Methods & clinical development, 2018-09, Vol.10, p.268-280
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Safety of CD34+ Hematopoietic Stem Cells and CD4+ T Lymphocytes Transduced with LVsh5/C46 in HIV-1 Infected Patients with High-Risk Lymphoma
Author / Creator:Delville, Marianne ; Touzot, Fabien ; Couzin, Chloé ; Hmitou, Isabelle ; Djerroudi, Lounes ; Ouedrani, Amani ; Lefrère, François ; Tuchman-Durand, Caroline ; Mollet, Chloé ; Fabreguettes, Jean-Roch ; Ferry, Nicolas ; Laganier, Laurent ; Magnani, Alessandra ; Magrin, Elisa ; Jolaine, Valérie ; Saez-Cirion, Asier ; Wolstein, Orit ; Symonds, Geoffrey ; Frange, Pierre ; Moins-Teisserenc, Hélène ; Chaix-Baudier, Marie-Laure ; Toubert, Antoine ; Larghero, Jérôme ; Parquet, Nathalie ; Brignier, Anne C. ; Barré-Sinoussi, Françoise ; Oksenhendler, Eric ; Cavazzana, Marina
Publisher:United States: Elsevier Inc
Journal title:Molecular therapy. Methods & clinical development, 2019-06, Vol.13, p.303-309
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279. Clinical Outcomes of Gene Therapy with BB305 Lentiviral Vector for Sickle Cell Disease and β-Thalassemia
Author / Creator:Cavazzana, Marina ; Ribeil, Jean-Antoine ; Payen, Emmanuel ; Touzot, Fabien ; Neven, Bénédicte ; Lefrere, Francois ; Suarez, Felipe ; Magrin, Elisa ; Beuzard, Yves ; Chretien, Stany ; Bourget, Philippe ; Monpoux, Fabrice ; Pondarré, Corinne ; Bartolucci, Pablo ; Schmidt, Manfred ; von Kalle, Christof ; Sandler, Laura ; Soni, Sandeep ; Semeraro, Michaela ; Nemer, Wassim El ; Hermine, Olivier ; de Montalembert, Mariane ; Blanche, Stéphane ; Hacein-Bey-Abina, Salima ; Leboulch, Philippe
Publisher:Elsevier Inc
Journal title:Molecular therapy, 2016-05, Vol.24, p.S111-S112
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An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype
Author / Creator:Leslie Weber ; Valentina Poletti ; Elisa Magrin ; Chiara Antoniani ; Samia Martin ; Charles Bayard ; Hanem Sadek ; Tristan Felix ; Vasco Meneghini ; Michael N. Antoniou ; Wassim El-Nemer ; Fulvio Mavilio ; Marina Cavazzana ; Isabelle Andre-Schmutz ; Annarita Miccio
Publisher:Elsevier
Journal title:Molecular therapy. Methods & clinical development, 2018-09, Vol.10, p.268-280
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Safety of CD34+ Hematopoietic Stem Cells and CD4+ T Lymphocytes Transduced with LVsh5/C46 in HIV-1 Infected Patients with High-Risk Lymphoma Safety of CD34+ Hematopoietic Stem Cells and CD4+ T Lymphoc...
Author / Creator:Marianne Delville ; Fabien Touzot ; Chloé Couzin ; Isabelle Hmitou ; Lounes Djerroudi ; Amani Ouedrani ; François Lefrère ; Caroline Tuchman-Durand ; Chloé Mollet ; Jean-Roch Fabreguettes ; Nicolas Ferry ; Laurent Laganier ; Alessandra Magnani ; Elisa Magrin ; Valérie Jolaine ; Asier Saez-Cirion ; Orit Wolstein ; Geoffrey Symonds ; Pierre Frange ; Hélène Moins-Teisserenc ; Marie-Laure Chaix-Baudier ; Antoine Toubert ; Jérôme Larghero ; Nathalie Parquet ; Anne C. Brignier ; Françoise Barré-Sinoussi ; Eric Oksenhendler ; Marina Cavazzana
Publisher:Elsevier
Journal title:Molecular therapy. Methods & clinical development, 2019-06, Vol.13, p.303-309
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Gene Therapy in a Patient with Sickle Cell Disease

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Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial

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Correction to: Ex vivo generated human T-lymphoid progenitors as a tool to accelerate immune reconstitution after partially HLA compatible hematopoietic stem cell transplantation or after gene therapy

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Severe inflammation and lineage skewing are associated with poor engraftment of engineered hematopoietic stem cells in patients with sickle cell disease

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Ex vivo generated human T-lymphoid progenitors as a tool to accelerate immune reconstitution after partially HLA compatible hematopoietic stem cell transplantation or after gene therapy

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559. Induction of Fetal Hemoglobin in Adult Erythroblasts by Genome Editing of the Beta-Globin Locus

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Gene Therapy in Patients with Transfusion-Dependent [beta]-Thalassemia

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231. Mixed Chimerism After Allogeneic Hematopoietic Stem Cell Transplantation in Sickle Cell Disease: Preliminary Results on Peripheral Blood Sorted Subpopulations and Erythroid Progenitors

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754. Exploring the Human Hematopoietic Hierarchy Through Retroviral Integration Sites Tracking in the Wiskott Aldrich Syndrome Gene Therapy Trial

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An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype

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Safety of CD34+ Hematopoietic Stem Cells and CD4+ T Lymphocytes Transduced with LVsh5/C46 in HIV-1 Infected Patients with High-Risk Lymphoma

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279. Clinical Outcomes of Gene Therapy with BB305 Lentiviral Vector for Sickle Cell Disease and β-Thalassemia

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An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype

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Safety of CD34+ Hematopoietic Stem Cells and CD4+ T Lymphocytes Transduced with LVsh5/C46 in HIV-1 Infected Patients with High-Risk Lymphoma Safety of CD34+ Hematopoietic Stem Cells and CD4+ T Lymphoc...

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