Autologous gene therapy for hemoglobinopathies: From bench to patient’s bedside
Locatelli, Franco, Cavazzana, Marina, Frangoul, Haydar, Fuente, Josu de la, Algeri, Mattia, Meisel, Roland
Published in Molecular therapy (01.05.2024)
Published in Molecular therapy (01.05.2024)
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Journal Article
Sickle Cell Disease: From Genetics to Curative Approaches
Hardouin, Giulia, Magrin, Elisa, Corsia, Alice, Cavazzana, Marina, Miccio, Annarita, Semeraro, Michaela
Published in Annual review of genomics and human genetics (25.08.2023)
Published in Annual review of genomics and human genetics (25.08.2023)
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Two Is Better Than One: Fetal Hemoglobin Reactivation and Alpha-Globin Downregulation to Correct the β-Hemoglobinopathy Phenotype through Base Editing
Antoniou, Panagiotis, von Joest, Mathieu, Fontana, Letizia, Hardouin, Giulia, Martinucci, Pierre, Cavazzana, Marina, Miccio, Annarita
Published in Blood (15.11.2022)
Published in Blood (15.11.2022)
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Journal Article
Adenine base editor–mediated correction of the common and severe IVS1-110 (G>A) β-thalassemia mutation
Hardouin, Giulia, Antoniou, Panagiotis, Martinucci, Pierre, Felix, Tristan, Manceau, Sandra, Joseph, Laure, Masson, Cécile, Scaramuzza, Samantha, Ferrari, Giuliana, Cavazzana, Marina, Miccio, Annarita
Published in Blood (09.03.2023)
Published in Blood (09.03.2023)
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Journal Article
Gene Therapy in a Patient with Sickle Cell Disease
Badat, Mohsin, Davies, James, Leboulch, Philippe, Cavazzana, Marina
Published in The New England journal of medicine (25.05.2017)
Published in The New England journal of medicine (25.05.2017)
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Journal Article
Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human b-globin locus
Antoniani, Chiara, Meneghini, Vasco, Lattanzi, Annalisa, Felix, Tristan, Romano, Oriana, Magrin, Elisa, Weber, Leslie, Pavani, Giulia, El Hoss, Sara, Kurita, Ryo, Nakamura, Yukio, Cradick, Thomas, Lundberg, Ante, Porteus, Matthew, Amendola, Mario, El Nemer, Wassim, Cavazzana, Marina, Mavilio, Fulvio, Miccio, Annarita
Published in Blood (26.04.2018)
Published in Blood (26.04.2018)
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Journal Article
A human immunodeficiency caused by mutations in the PIK3R1 gene
Deau, Marie-Céline, Heurtier, Lucie, Frange, Pierre, Suarez, Felipe, Bole-Feysot, Christine, Nitschke, Patrick, Cavazzana, Marina, Picard, Capucine, Durandy, Anne, Fischer, Alain, Kracker, Sven
Published in The Journal of clinical investigation (01.09.2014)
Published in The Journal of clinical investigation (01.09.2014)
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Journal Article
Blood diseases in Africa: Redressing unjust disparities is an urgent unmet need
Makani, Julie, Cavazzana, Marina, Gupta, Kalpna, Nnodu, Obiageli, Odame, Isaac, Tshilolo, Leon, Ware, Russell, Luzzatto, Lucio
Published in American journal of hematology (01.12.2022)
Published in American journal of hematology (01.12.2022)
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Journal Article
Access to gene therapy for rare diseases when commercialization is not fit for purpose
Fox, Thomas, Bueren, Juan, Candotti, Fabio, Fischer, Alain, Aiuti, Alessandro, Lankester, Arjan, Booth, Claire
Published in Nature medicine (01.03.2023)
Published in Nature medicine (01.03.2023)
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Severe combined immunodeficiency and microcephaly in siblings with hypomorphic mutations in DNA ligase IV
Buck, Dietke, Moshous, Despina, de Chasseval, Régina, Ma, Yunmei, le Deist, Françoise, Cavazzana‐Calvo, Marina, Fischer, Alain, Casanova, Jean‐Laurent, Lieber, Michael R., de Villartay, Jean‐Pierre
Published in European Journal of Immunology (01.01.2006)
Published in European Journal of Immunology (01.01.2006)
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Journal Article
Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia
Thompson, Alexis A, Walters, Mark C, Kwiatkowski, Janet, Rasko, John E.J, 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
Published in The New England journal of medicine (19.04.2018)
Published in The New England journal of medicine (19.04.2018)
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Journal Article
Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β(A(T87Q))-Globin Gene
Negre, Olivier, Eggimann, Anne-Virginie, Beuzard, Yves, Ribeil, Jean-Antoine, Bourget, Philippe, Borwornpinyo, Suparerk, Hongeng, Suradej, Hacein-Bey, Salima, Cavazzana, Marina, Leboulch, Philippe, Payen, Emmanuel
Published in Human gene therapy (01.02.2016)
Published in Human gene therapy (01.02.2016)
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Journal Article
Novel Lentiviral Vectors for Gene Therapy of Sickle Cell Disease Combining Gene Addition and Gene Silencing Strategies
Brusson, Megane, Chalumeau, Anne, Martinucci, Pierre, Poletti, Valentina, Mavilio, Fulvio, Cavazzana, Marina, Amendola, Mario, Miccio, Annarita
Published in Blood (23.11.2021)
Published in Blood (23.11.2021)
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Journal Article
Sickle Cell Disease: From Genetics to Curative Approaches
Hardouin, Giulia, Magrin, Elisa, Corsia, Alice, Cavazzana, Marina, Miccio, Annarita, Semeraro, Michaela
Published in Annual review of genomics and human genetics (01.01.2023)
Published in Annual review of genomics and human genetics (01.01.2023)
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Journal Article
A specific time course for mobilization of peripheral blood CD34+ cells after plerixafor injection in very poor mobilizer patients: impact on the timing of the apheresis procedure
Lefrère, François, Mauge, Laeticia, Réa, Delphine, Ribeil, Jean-Antoine, Dal Cortivo, Liliane, Brignier, Anne C., Aoun, Charbel, Larghéro, Jérôme, Cavazzana-Calvo, Marina, Micléa, Jean-Michel
Published in Transfusion (Philadelphia, Pa.) (01.03.2013)
Published in Transfusion (Philadelphia, Pa.) (01.03.2013)
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Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation
Gluckman, Eliane, Cappelli, Barbara, Bernaudin, Francoise, Labopin, Myriam, Volt, Fernanda, Carreras, Jeanette, Pinto Simões, Belinda, Ferster, Alina, Dupont, Sophie, de la Fuente, Josu, Dalle, Jean-Hugues, Zecca, Marco, Walters, Mark C., Krishnamurti, Lakshmanan, Bhatia, Monica, Leung, Kathryn, Yanik, Gregory, Kurtzberg, Joanne, Dhedin, Nathalie, Kuentz, Mathieu, Michel, Gerard, Apperley, Jane, Lutz, Patrick, Neven, Bénédicte, Bertrand, Yves, Vannier, Jean Pierre, Ayas, Mouhab, Cavazzana, Marina, Matthes-Martin, Susanne, Rocha, Vanderson, Elayoubi, Hanadi, Kenzey, Chantal, Bader, Peter, Locatelli, Franco, Ruggeri, Annalisa, Eapen, Mary
Published in Blood (16.03.2017)
Published in Blood (16.03.2017)
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Long-term follow-up of IPEX syndrome patients after different therapeutic strategies: An international multicenter retrospective study
Barzaghi, Federica, Amaya Hernandez, Laura Cristina, Neven, Benedicte, Ricci, Silvia, Kucuk, Zeynep Yesim, Bleesing, Jack J., Nademi, Zohreh, Slatter, Mary Anne, Ulloa, Erlinda Rose, Shcherbina, Anna, Roppelt, Anna, Worth, Austen, Silva, Juliana, Aiuti, Alessandro, Murguia-Favela, Luis, Speckmann, Carsten, Carneiro-Sampaio, Magda, Fernandes, Juliana Folloni, Baris, Safa, Ozen, Ahmet, Karakoc-Aydiner, Elif, Kiykim, Ayca, Schulz, Ansgar, Steinmann, Sandra, Notarangelo, Lucia Dora, Gambineri, Eleonora, Lionetti, Paolo, Shearer, William Thomas, Forbes, Lisa R., Martinez, Caridad, Moshous, Despina, Blanche, Stephane, Fisher, Alain, Ruemmele, Frank M., Tissandier, Come, Ouachee-Chardin, Marie, Rieux-Laucat, Frédéric, Cavazzana, Marina, Qasim, Waseem, Lucarelli, Barbarella, Albert, Michael H., Kobayashi, Ichiro, Alonso, Laura, Diaz De Heredia, Cristina, Kanegane, Hirokazu, Lawitschka, Anita, Seo, Jong Jin, Gonzalez-Vicent, Marta, Diaz, Miguel Angel, Goyal, Rakesh Kumar, Sauer, Martin G., Yesilipek, Akif, Kim, Minsoo, Yilmaz-Demirdag, Yesim, Bhatia, Monica, Khlevner, Julie, Richmond Padilla, Erick J., Martino, Silvana, Montin, Davide, Neth, Olaf, Molinos-Quintana, Agueda, Valverde-Fernandez, Justo, Broides, Arnon, Pinsk, Vered, Ballauf, Antje, Haerynck, Filomeen, Bordon, Victoria, Dhooge, Catharina, Garcia-Lloret, Maria Laura, Bredius, Robbert G., Kałwak, Krzysztof, Haddad, Elie, Seidel, Markus Gerhard, Duckers, Gregor, Pai, Sung-Yun, Dvorak, Christopher C., Ehl, Stephan, Locatelli, Franco, Goldman, Frederick, Gennery, Andrew Richard, Cowan, Mort J., Roncarolo, Maria-Grazia, Bacchetta, Rosa
Published in Journal of allergy and clinical immunology (01.03.2018)
Published in Journal of allergy and clinical immunology (01.03.2018)
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International AIDS Society global scientific strategy: towards an HIV cure 2016
Deeks, Steven G, Lewin, Sharon R, Ross, Anna Laura, Ananworanich, Jintanat, Benkirane, Monsef, Cannon, Paula, Chomont, Nicolas, Douek, Daniel, Lifson, Jeffrey D, Lo, Ying-Ru, Kuritzkes, Daniel, Margolis, David, Mellors, John, Persaud, Deborah, Tucker, Joseph D, Barre-Sinoussi, Françoise, Alter, Galit, Auerbach, Judith, Autran, Brigitte, Barouch, Dan H, Behrens, Georg, Cavazzana, Marina, Chen, Zhiwei, Cohen, Éric A, Corbelli, Giulio Maria, Eholié, Serge, Eyal, Nir, Fidler, Sarah, Garcia, Laurindo, Grossman, Cynthia, Henderson, Gail, Henrich, Timothy J, Jefferys, Richard, Kiem, Hans-Peter, McCune, Joseph, Moodley, Keymanthri, Newman, Peter A, Nijhuis, Monique, Nsubuga, Moses Supercharger, Ott, Melanie, Palmer, Sarah, Richman, Douglas, Saez-Cirion, Asier, Sharp, Matthew, Siliciano, Janet, Silvestri, Guido, Singh, Jerome, Spire, Bruno, Taylor, Jeffrey, Tolstrup, Martin, Valente, Susana, van Lunzen, Jan, Walensky, Rochelle, Wilson, Ira, Zack, Jerome
Published in Nature Medicine (01.08.2016)
Published in Nature Medicine (01.08.2016)
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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
Published in JAMA : the journal of the American Medical Association (21.04.2015)
Published in JAMA : the journal of the American Medical Association (21.04.2015)
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