Marina Cavazzana (M Cavazzana)

Publication Activity (10 Years)

Years Active: 2017-2024
Publications (10 Years): 31

Top Topics

Top Venues

The Journal of experimental medicine

Nature communications

This page only lists publications with an associated author ORCID identifier.

Publications

Nataël Sorel, Francisco Díaz-Pascual, Boris Bessot, Hanem Sadek, Chloé Mollet, Myriam Chouteau, Marco Zahn, Irene Gil-Farina, Parisa Tajer, Marja van Eggermond, Dagmar Berghuis, Arjan C Lankester, Isabelle André, Richard Gabriel, Marina Cavazzana, Kasrin Pike-Overzet, Frank J T Staal, Chantal Lagresle-Peyrou
Restoration of T and B Cell Differentiation after RAG1 Gene Transfer in Human RAG1 Defective Hematopoietic Stem Cells. Biomedicines 12 (7) (2024)
Arianna Moiani, Gil Letort, Sabrina Lizot, Anne Chalumeau, Chloe Foray, Tristan Felix, Diane Le Clerre, Sonal Temburni, Patrick Hong, Sophie Leduc, Noemie Pinard, Alan Marechal, Eduardo Seclen, Alex Boyne, Louisa Mayer, Robert Hong, Sylvain Pulicani, Roman Galetto, Agnès Gouble, Marina Cavazzana, Alexandre Juillerat, Annarita Miccio, Aymeric Duclert, Philippe Duchateau, Julien Valton
Non-viral DNA delivery and TALEN editing correct the sickle cell mutation in hematopoietic stem cells. Nature communications 15 (1) (2024)
Arianna Moiani, Gil Letort, Sabrina Lizot, Anne Chalumeau, Chloe Foray, Tristan Felix, Diane Le Clerre, Sonal Temburni, Patrick Hong, Sophie Leduc, Noemie Pinard, Alan Marechal, Eduardo Seclen, Alex Boyne, Louisa Mayer, Robert Hong, Sylvain Pulicani, Roman Galetto, Agnès Gouble, Marina Cavazzana, Alexandre Juillerat, Annarita Miccio, Aymeric Duclert, Philippe Duchateau, Julien Valton
Non-viral DNA delivery and TALEN editing correct the sickle cell mutation in hematopoietic stem cells. Nature communications 15 (1) (2024)
Nataël Sorel, Francisco Díaz-Pascual, Boris Bessot, Hanem Sadek, Chloé Mollet, Myriam Chouteau, Marco Zahn, Irene Gil-Farina, Parisa Tajer, Marja van Eggermond, Dagmar Berghuis, Arjan C Lankester, Isabelle André, Richard Gabriel, Marina Cavazzana, Kasrin Pike-Overzet, Frank J T Staal, Chantal Lagresle-Peyrou
Restoration of T and B Cell Differentiation after RAG1 Gene Transfer in Human RAG1 Defective Hematopoietic Stem Cells. Biomedicines 12 (7) (2024)
Anne Doye, Paul Chaintreuil, Chantal Lagresle-Peyrou, Ludovic Batistic, Valentine Marion, Patrick Munro, Celine Loubatier, Rayana Chirara, Nataël Sorel, Boris Bessot, Pauline Bronnec, Julie Contenti, Johan Courjon, Valerie Giordanengo, Arnaud Jacquel, Pascal Barbry, Marie Couralet, Nathalie Aladjidi, Alain Fischer, Marina Cavazzana, Coralie Mallebranche, Orane Visvikis, Sven Kracker, Despina Moshous, Els Verhoeyen, Laurent Boyer
RAC2 gain-of-function variants causing inborn error of immunity drive NLRP3 inflammasome activation. The Journal of experimental medicine 221 (10) (2024)
Nataël Sorel, Francisco Díaz-Pascual, Boris Bessot, Hanem Sadek, Chloé Mollet, Myriam Chouteau, Marco Zahn, Irene Gil-Farina, Parisa Tajer, Marja van Eggermond, Dagmar Berghuis, Arjan C Lankester, Isabelle André, Richard Gabriel, Marina Cavazzana, Kasrin Pike-Overzet, Frank J T Staal, Chantal Lagresle-Peyrou
Restoration of T and B Cell Differentiation after RAG1 Gene Transfer in Human RAG1 Defective Hematopoietic Stem Cells. Biomedicines 12 (7) (2024)
Arianna Moiani, Gil Letort, Sabrina Lizot, Anne Chalumeau, Chloe Foray, Tristan Felix, Diane Le Clerre, Sonal Temburni, Patrick Hong, Sophie Leduc, Noemie Pinard, Alan Marechal, Eduardo Seclen, Alex Boyne, Louisa Mayer, Robert Hong, Sylvain Pulicani, Roman Galetto, Agnès Gouble, Marina Cavazzana, Alexandre Juillerat, Annarita Miccio, Aymeric Duclert, Philippe Duchateau, Julien Valton
Non-viral DNA delivery and TALEN editing correct the sickle cell mutation in hematopoietic stem cells. Nature communications 15 (1) (2024)
Romane Thouenon, Loïc Chentout, Nidia Carolina Moreno-Corona, Lucie Poggi, Emilia Puig Lombardi, Bénédicte Hoareau-Coudert, Yohann Schmitt, Chantal Lagresle-Peyrou, Jacinta Bustamante, Isabelle André-Schmutz, Marina Cavazzana, Anne Durandy, Jean Laurent Casanova, Lionel Galicier, Jehane Fadlallah, Alain Fischer, Sven Kracker
A neomorphic mutation in the interferon activation domain of IRF4 causes a dominant primary immunodeficiency. The Journal of experimental medicine 220 (6) (2023)
Romane Thouenon, Loïc Chentout, Nidia Carolina Moreno-Corona, Lucie Poggi, Emilia Puig Lombardi, Bénédicte Hoareau-Coudert, Yohann Schmitt, Chantal Lagresle-Peyrou, Jacinta Bustamante, Isabelle André-Schmutz, Marina Cavazzana, Anne Durandy, Jean Laurent Casanova, Lionel Galicier, Jehane Fadlallah, Alain Fischer, Sven Kracker
A neomorphic mutation in the interferon activation domain of IRF4 causes a dominant primary immunodeficiency. The Journal of experimental medicine 220 (6) (2023)
Romane Thouenon, Loïc Chentout, Nidia Carolina Moreno-Corona, Lucie Poggi, Emilia Puig Lombardi, Bénédicte Hoareau-Coudert, Yohann Schmitt, Chantal Lagresle-Peyrou, Jacinta Bustamante, Isabelle André-Schmutz, Marina Cavazzana, Anne Durandy, Jean Laurent Casanova, Lionel Galicier, Jehane Fadlallah, Alain Fischer, Sven Kracker
A neomorphic mutation in the interferon activation domain of IRF4 causes a dominant primary immunodeficiency. The Journal of experimental medicine 220 (6) (2023)
Laure Joseph, Sandra Manceau, Didier Borderie, Catherine Patrat, Jean Benoit Arlet, Benoit Meunier, Marina Cavazzana, Pietro Santulli, Virginie Barraud-Lange
Detrimental effects of sickle cell disease and hydroxycarbamide on ovarian reserve but uncertain impact on fertility. Blood advances (2023)
Giulia Hardouin, Panagiotis Antoniou, Pierre Martinucci, Tristan Felix, Sandra Manceau, Laure Joseph, Cecile Masson, Samantha Scaramuzza, Giuliana Ferrari, Marina Cavazzana, Annarita Miccio
Adenine base editor-mediated correction of the common and severe IVS1-110 (G>A) β-thalassemia mutation. Blood 141 (10) (2023)
Giulia Hardouin, Panagiotis Antoniou, Pierre Martinucci, Tristan Felix, Sandra Manceau, Laure Joseph, Cecile Masson, Samantha Scaramuzza, Giuliana Ferrari, Marina Cavazzana, Annarita Miccio
Adenine base editor-mediated correction of the common and severe IVS1-110 (G>A) β-thalassemia mutation. Blood 141 (10) (2023)
Romane Thouenon, Loïc Chentout, Nidia Carolina Moreno-Corona, Lucie Poggi, Emilia Puig Lombardi, Bénédicte Hoareau-Coudert, Yohann Schmitt, Chantal Lagresle-Peyrou, Jacinta Bustamante, Isabelle André-Schmutz, Marina Cavazzana, Anne Durandy, Jean Laurent Casanova, Lionel Galicier, Jehane Fadlallah, Alain Fischer, Sven Kracker
A neomorphic mutation in the interferon activation domain of IRF4 causes a dominant primary immunodeficiency. The Journal of experimental medicine 220 (6) (2023)
Romane Thouenon, Loïc Chentout, Nidia Carolina Moreno-Corona, Lucie Poggi, Emilia Puig Lombardi, Bénédicte Hoareau-Coudert, Yohann Schmitt, Chantal Lagresle-Peyrou, Jacinta Bustamante, Isabelle André-Schmutz, Marina Cavazzana, Anne Durandy, Jean Laurent Casanova, Lionel Galicier, Jehane Fadlallah, Alain Fischer, Sven Kracker
A neomorphic mutation in the interferon activation domain of IRF4 causes a dominant primary immunodeficiency. The Journal of experimental medicine 220 (6) (2023)
Elisa Magrin, Michaela Semeraro, Nicolas Hebert, Laure Joseph, Alessandra Magnani, Anne Chalumeau, Aurélie Gabrion, Cécile Roudaut, Jouda Marouene, Francois Lefrere, Jean-Sébastien Diana, Adeline Denis, Bénédicte Neven, Isabelle Funck-Brentano, Olivier Negre, Sylvain Renolleau, Valentine Brousse, Laurent Kiger, Fabien Touzot, Catherine Poirot, Philippe Bourget, Wassim El Nemer, Stéphane Blanche, Jean-Marc Tréluyer, Mohammed Asmal, Courtney Walls, Yves Beuzard, Manfred Schmidt, Salima Hacein-Bey-Abina, Vahid Asnafi, Isabelle Guichard, Maryline Poirée, Fabrice Monpoux, Philippe Touraine, Chantal Brouzes, Mariane de Montalembert, Emmanuel Payen, Emmanuelle Six, Jean-Antoine Ribeil, Annarita Miccio, Pablo Bartolucci, Philippe Leboulch, Marina Cavazzana
Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial. Nature medicine 28 (1) (2022)
Michael H Albert, Mary A Slatter, Andrew R Gennery, Tayfun Güngör, Katerina Bakunina, Benyamin Markovitch, Sheree Hazelaar, Tiarlan Sirait, Virginie Courteille, Alessandro Aiuti, Olga V Aleinikova, Dmitry Balashov, Maria Ester Bernardo, Ivana Bodova, Benedicte Bruno, Marina Cavazzana, Robert Chiesa, Alain Fischer, Fabian H Hauck, Marianne Ifversen, Krzyzstof Kalwak, Christoph Klein, Alexander Dmitrievich Kulagin, Alphan Kupesiz, Bulent Baris Kuskonmaz, Caroline A Lindemans, Franco Locatelli, Su Han Lum, Alexey A Maschan, Richard P Meisel, Despina Moshous, Fulvio Porta, Martin G Sauer, Petr Sedlacek, Ansgar Schulz, Felipe Suarez, Tanja C Vallée, Jacek H Winiarski, Marco Zecca, Bénédicte Neven, Paul Veys, Arjan C Lankester
Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT Inborn Errors Working Party analysis. Blood 139 (13) (2022)
Giulia Hardouin, Panagiotis Antoniou, Pierre Martinucci, Tristan Felix, Sandra Manceau, Laure Joseph, Cecile Masson, Samantha Scaramuzza, Giuliana Ferrari, Marina Cavazzana, Annarita Miccio
Adenine base editor-mediated correction of the common and severe IVS1-110 (G>A) β-thalassemia mutation. Blood (2022)
Panagiotis Antoniou, Giulia Hardouin, Pierre Martinucci, Giacomo Frati, Tristan Felix, Anne Chalumeau, Letizia Fontana, Jeanne Martin, Cecile Masson, Megane Brusson, Giulia Maule, Marion Rosello, Carine Giovannangeli, Vincent Abramowski, Jean Pierre de Villartay, Jean-Paul Concordet, Filippo Del Bene, Wassim El Nemer, Mario Amendola, Marina Cavazzana, Anna Cereseto, Oriana Romano, Annarita Miccio
Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression. Nature communications 13 (1) (2022)
Giulia Hardouin, Panagiotis Antoniou, Pierre Martinucci, Tristan Felix, Sandra Manceau, Laure Joseph, Cecile Masson, Samantha Scaramuzza, Giuliana Ferrari, Marina Cavazzana, Annarita Miccio
Adenine base editor-mediated correction of the common and severe IVS1-110 (G>A) β-thalassemia mutation. Blood (2022)
Clément Pontoizeau, Marcelo Simon-Sola, Clovis Gaborit, Vincent Nguyen, Irina Rotaru, Nolan Tual, Pasqualina Colella, Muriel Girard, Maria-Grazia Biferi, Jean-Baptiste Arnoux, Agnes Rotig, Chris Ottolenghi, Pascale de Lonlay, Federico Mingozzi, Marina Cavazzana, Manuel Schiff
Neonatal gene therapy achieves sustained disease rescue of maple syrup urine disease in mice. Nature communications 13 (1) (2022)
A Magnani, M Semeraro, Frédéric Adam, C Booth, L Dupré, Emma Catherine Morris, A Gabrion, C Roudaut, D Borgel, Antoine Toubert, Emmanuel Clave, C Abdo, G Gorochov, R Petermann, M Guiot, M Miyara, D Moshous, E Magrin, A Denis, F Suarez, Chantal Lagresle-Peyrou, A M Roche, J Everett, A Trinquand, M Guisset, J Xu Bayford, S Hacein-Bey-Abina, Alexandre Kauskot, R Elfeky, C Rivat, S Abbas, H B Gaspar, Elizabeth A Macintyre, C Picard, Frederic D Bushman, A Galy, Alain Fischer, Emmanuelle Six, Adrian J Thrasher, Marina Cavazzana
Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott-Aldrich syndrome. Nature medicine 28 (1) (2022)
Catharina Schuetz, Julia Gerke, Markus Johannes Ege, Jolan Eszter Walter, Maaike A A Kusters, Austen J J Worth, Jennifer A Kanakry, Dimana Dimitrova, Beata Wolska-Kusnierz, Karin Chen, Ekrem Unal, Musa Karakukcu, Olga Pashchenko, Jennifer W Leiding, Tomoki Kawai, Persis Jal Amrolia, Dagmar Berghuis, Jochen Buechner, David Buchbinder, Morton J Cowan, Andrew R Gennery, Tayfun Güngör, Jennifer R Heimall, Maurizio Miano, Isabelle Meyts, Emma Catherine Morris, Jacques G Riviere, Svetlana O Sharapova, Peter J Shaw, Mary A Slatter, Manfred Honig, Paul Veys, Alain Fischer, Marina Cavazzana, Despina Moshous, Ansgar S Schulz, Michael H Albert, Jennifer M Puck, Arjan C Lankester, Luigi Daniele Notarangelo, Bénédicte Neven
Hypomorphic RAG deficiency: impact of disease burden on survival and thymic recovery argues for early diagnosis and HSCT. Blood (2022)
Marina Cavazzana
Four decades of progress. Blood 140 (7) (2022)
Baptiste Lamarthée, Armance Marchal, Soëli Charbonnier, Tifanie Blein, Juliette Léon, Emmanuel Martin, Lucas Rabaux, Katrin Vogt, Matthias Titeux, Marianne Delville, Hélène Vinçon, Emmanuelle Six, Nicolas Pallet, David Michonneau, Dany Anglicheau, Christophe Legendre, Jean-Luc Taupin, Ivan Nemazanyy, Birgit Sawitzki, Sylvain Latour, Marina Cavazzana, Isabelle André-Schmutz, Julien Zuber
Transient mTOR inhibition rescues 4-1BB CAR-Tregs from tonic signal-induced dysfunction. Nature communications 12 (1) (2021)
Leslie Weber, Giacomo Frati, Tristan Felix, Giulia Hardouin, Antonio Casini, Clara Wollenschlaeger, Vasco Meneghini, Cecile Masson, Anne De Cian, Anne Chalumeau, Fulvio Mavilio, Mario Amendola, Isabelle Andre-Schmutz, Anna Cereseto, Wassim El Nemer, Jean-Paul Concordet, Carine Giovannangeli, Marina Cavazzana, Annarita Miccio
Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype. Science advances 6 (7) (2020)
Nieves Diaz, Maria Juarez, Caterina Cancrini, Maximilian Heeg, Pere Soler-Palacin, Andrew Payne, Geoffrey I Johnston, Eric Helmer, Dionne Cain, Joanne Mann, Daisy Yuill, Francesca Conti, Silvia Di Cesare, Stephan Ehl, Marina Garcia-Prat, Maria Elena Maccari, Andrea Martin-Nalda, Mónica Martínez-Gallo, Despina Moshous, Veronica Santilli, Michaela Semeraro, Alessandra Simonetti, Felipe Suarez, Marina Cavazzana, Sven Kracker
Seletalisib for Activated PI3Kδ Syndromes: Open-Label Phase 1b and Extension Studies. Journal of immunology (Baltimore, Md. : 1950) 205 (11) (2020)
Julien Zuber, Olivia Boyer, Bénédicte Neven, Isabelle Jollet, Virginie Renac, Romain Berthaud, Romain Levy, Baptiste Lamarthée, Jonathan Visentin, Armance Marchal, Nathalie Gouge-Biebuyck, Astrid Godron-Dubrasquet, Nathalie Aladjidi, Melissa O Rabah, Sarah Winter, Juliette Léon, Michael Dussiot, Marion Rabant, Saoussen Krid, Pauline Krug, Marina Charbit, Florence Lacaille, Isabelle André-Schmutz, Marina Cavazzana, Brigitte Llanas, Lise Allard, Françoise Pirenne, Sylvie Gross, Rachid Djoudi, Pierre Tiberghien, Jean-Luc Taupin, Stéphane Blanche, Rémi Salomon
Donor-targeted serotherapy as a rescue therapy for steroid-resistant acute GVHD after HLA-mismatched kidney transplantation. American journal of transplantation : official journal of the American Society of Transplantation and the American Society of Transplant Surgeons 20 (8) (2020)
Marina Cavazzana, Frederic D Bushman, Annarita Miccio, Isabelle André-Schmutz, Emmanuelle Six
Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges. Nature reviews. Drug discovery 18 (6) (2019)
Chiara Antoniani, Vasco Meneghini, Annalisa Lattanzi, Tristan Felix, Oriana Romano, Elisa Magrin, Leslie Weber, Giulia Pavani, Sara El Hoss, Ryo Kurita, Yukio Nakamura, Thomas J Cradick, Ante S Lundberg, Matthew H Porteus, Mario Amendola, Wassim El Nemer, Marina Cavazzana, Fulvio Mavilio, Annarita Miccio
Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus. Blood 131 (17) (2018)
Tayebeh Soheili, Amandine Durand, Fernando E Sepulveda, Julie Rivière, Chantal Lagresle-Peyrou, Hanem Sadek, Geneviève de Saint Basile, Samia Martin, Fulvio Mavilio, Marina Cavazzana, Isabelle André-Schmutz
Gene transfer into hematopoietic stem cells reduces HLH manifestations in a murine model of Munc13-4 deficiency. Blood advances 1 (27) (2017)