Gene Therapy in a Patient with Sickle Cell Disease
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
Published in The New England journal of medicine (02.03.2017)
Published in The New England journal of medicine (02.03.2017)
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Transient mTOR inhibition rescues 4-1BB CAR-Tregs from tonic signal-induced dysfunction
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Published in Nature communications (08.11.2021)
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Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial
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Published in Nature medicine (01.01.2022)
Published in Nature medicine (01.01.2022)
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Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel
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Published in Haematologica (Roma) (01.05.2014)
Published in Haematologica (Roma) (01.05.2014)
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Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem cells in sickle cell disease patients after exchange transfusion
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Published in Haematologica (Roma) (01.05.2018)
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Retrieval of vector integration sites from cell-free DNA
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Published in Nature medicine (01.08.2021)
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Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression
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Published in Nature communications (04.11.2022)
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An in vivo genetic reversion highlights the crucial role of Myb-Like, SWIRM, and MPN domains 1 (MYSM1) in human hematopoiesis and lymphocyte differentiation
Le Guen, Tangui, PhD, Touzot, Fabien, MD, PhD, André-Schmutz, Isabelle, PhD, Lagresle-Peyrou, Chantal, PhD, France, Benoit, MSc, Kermasson, Laetitia, Lambert, Nathalie, Picard, Capucine, MD, PhD, Nitschke, Patrick, PhD, Carpentier, Wassila, PhD, Bole-Feysot, Christine, PhD, Lim, Annick, PhD, Cavazzana, Marina, MD, PhD, Callebaut, Isabelle, PhD, Soulier, Jean, MD, PhD, Jabado, Nada, MD, PhD, Fischer, Alain, MD, PhD, de Villartay, Jean-Pierre, PhD, Revy, Patrick, PhD
Published in Journal of allergy and clinical immunology (01.12.2015)
Published in Journal of allergy and clinical immunology (01.12.2015)
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Published in Nature immunology (01.05.2021)
Published in Nature immunology (01.05.2021)
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Autologous Stem-Cell-Based Gene Therapy for Inherited Disorders: State of the Art and Perspectives
Staal, Frank J. T., Aiuti, Alessandro, Cavazzana, Marina
Published in Frontiers in pediatrics (31.10.2019)
Published in Frontiers in pediatrics (31.10.2019)
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Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: Entering a new century, do we do better?
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
Published in Journal of allergy and clinical immunology (01.09.2010)
Published in Journal of allergy and clinical immunology (01.09.2010)
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A gain-of-function RAC2 mutation is associated with bone-marrow hypoplasia and an autosomal dominant form of severe combined immunodeficiency
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Published in Haematologica (Roma) (01.02.2021)
Published in Haematologica (Roma) (01.02.2021)
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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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Published in Bone marrow transplantation (Basingstoke) (01.05.2022)
Published in Bone marrow transplantation (Basingstoke) (01.05.2022)
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Risk factors and outcomes according to age at transplantation with an HLA-identical sibling for sickle cell disease
Cappelli, Barbara, Volt, Fernanda, Tozatto-Maio, Karina, Scigliuolo, Graziana Maria, Ferster, Alina, Dupont, Sophie, Simões, Belinda Pinto, Al-Seraihy, Amal, Aljurf, Mahmoud D, Almohareb, Fahad, Belendez, Cristina, Matthes, Susanne, Dhedin, Nathalie, Pondarre, Corinne, Dalle, Jean-Hugues, Bertrand, Yves, Vannier, Jean Pierre, Kuentz, Mathieu, Lutz, Patrick, Michel, Gérard, Rafii, Hanadi, Neven, Benedicte, Zecca, Marco, Bader, Peter, Cavazzana, Marina, Labopin, Myriam, Locatelli, Franco, Magnani, Alessandra, Ruggeri, Annalisa, Rocha, Vanderson, Bernaudin, Françoise, de La Fuente, Josu, Corbacioglu, Selim, Gluckman, Eliane
Published in Haematologica (Roma) (01.12.2019)
Published in Haematologica (Roma) (01.12.2019)
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Non-viral DNA delivery and TALEN editing correct the sickle cell mutation in hematopoietic stem cells
Moiani, Arianna, Letort, Gil, Lizot, Sabrina, Chalumeau, Anne, Foray, Chloe, Felix, Tristan, Le Clerre, Diane, Temburni-Blake, Sonal, Hong, Patrick, Leduc, Sophie, Pinard, Noemie, Marechal, Alan, Seclen, Eduardo, Boyne, Alex, Mayer, Louisa, Hong, Robert, Pulicani, Sylvain, Galetto, Roman, Gouble, Agnès, Cavazzana, Marina, Juillerat, Alexandre, Miccio, Annarita, Duclert, Aymeric, Duchateau, Philippe, Valton, Julien
Published in Nature communications (11.06.2024)
Published in Nature communications (11.06.2024)
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