Innovations Needed for Effective Implementation of Ex Vivo Gene Therapies

por Cavazzana, Marina
Publicado 2017

Gene Therapy for Hemoglobinopathies

por Cavazzana, Marina, Mavilio, Fulvio
Publicado 2018

Gene therapy for Whiskott–Aldrich syndrome: The latest news

por Cavazzana, Marina, Thrasher, Adrian
Publicado 2022

Concise Review: Boosting T‐Cell Reconstitution Following Allogeneic Transplantation—Current Concepts and Future Perspectives

por Simons, Laura, Cavazzana, Marina, André, Isabelle
Publicado 2019

Autologous Stem-Cell-Based Gene Therapy for Inherited Disorders: State of the Art and Perspectives

por Staal, Frank J. T., Aiuti, Alessandro, Cavazzana, Marina
Publicado 2019

S265: MATERNAL AND PERINATAL OUTCOMES OF SICKLE CELL DISEASE IN PREGNANCY: A NATIONWIDE STUDY IN FRANCE.

por Corsia, Alice, Joseph, Laure, Beeker, Nathanael, Manceau, Sandra, Driessen, Marine, Cavazzana, Marina, Collier, Mathis, Marc Treluyer, Jean
Publicado 2023

Development of Lentiviral Vectors Simultaneously Expressing Multiple siRNAs Against CCR5, vif and tat/rev Genes for an HIV-1 Gene Therapy Approach

por Spanevello, Francesca, Calistri, Arianna, Del Vecchio, Claudia, Mantelli, Barbara, Frasson, Chiara, Basso, Giuseppe, Palù, Giorgio, Cavazzana, Marina, Parolin, Cristina
Publicado 2016

T-Cell Progenitors As A New Immunotherapy to Bypass Hurdles of Allogeneic Hematopoietic Stem Cell Transplantation

por Gaudeaux, Pierre, Moirangthem, Ranjita Devi, Bauquet, Aurélie, Simons, Laura, Joshi, Akshay, Cavazzana, Marina, Nègre, Olivier, Soheili, Shabi, André, Isabelle
Publicado 2022

Detrimental effects of sickle cell disease and hydroxycarbamide on ovarian reserve but uncertain impact on fertility

por Joseph, Laure, Manceau, Sandra, Borderie, Didier, Patrat, Catherine, Arlet, Jean Benoit, Meunier, Benoit, Cavazzana, Marina, Santulli, Pietro, Barraud-Lange, Virginie
Publicado 2023

A Nontoxic Transduction Enhancer Enables Highly Efficient Lentiviral Transduction of Primary Murine T Cells and Hematopoietic Stem Cells

por Delville, Marianne, Soheili, Tayebeh, Bellier, Florence, Durand, Amandine, Denis, Adeline, Lagresle-Peyrou, Chantal, Cavazzana, Marina, Andre-Schmutz, Isabelle, Six, Emmanuelle
Publicado 2018

PB2514: IMMUNOGENICITY OF ANTI-SARS-COV-2 VACCINES IN PATIENTS WITH SEVERE SICKLE CELL DISEASE

por Meunier, Benoit, Manceau, Sandra, Corsia, Alice, Ihlem, Rahal, Jestin, Mathhieu, Verheyden, Laetitia, Lefrère, François, Cavazzana, Marina, Arlet, Jean-Benoît, Joseph, Laure
Publicado 2023

Alternative UNC13D Promoter Encodes a Functional Munc13-4 Isoform Predominantly Expressed in Lymphocytes and Platelets

por Galgano, Donatella, Soheili, Tayebeh, Voss, Matthias, Torralba-Raga, Lamberto, Tesi, Bianca, Cichocki, Frank, Andre, Isabelle, Rettig, Jens, Cavazzana, Marina, Bryceson, Yenan
Publicado 2020

Adenine base editor–mediated correction of the common and severe IVS1-110 (G>A) β-thalassemia mutation

por Hardouin, Giulia, Antoniou, Panagiotis, Martinucci, Pierre, Felix, Tristan, Manceau, Sandra, Joseph, Laure, Masson, Cécile, Scaramuzza, Samantha, Ferrari, Giuliana, Cavazzana, Marina, Miccio, Annarita
Publicado 2023

S274: A NEW, EFFECTIVE, SAFE, NON-MYELOABLATIVE CONDITIONING REGIMEN WITH A HLA-MATCHED SIBLING DONOR FOR THE SEVERE SICKLE CELL PHENOTYPE IN ADULTS

por Ihlem, Rahal, Marçais, Ambroise, Cazelles, Clarisse, Manceau, Sandra, Ribeil, Jean-Antoine, Arlet, Jean-Benoît, Meunier, Benoit, Corsia, Alice, Cavazzana, Marina, Suarez, Felipe, Joseph, Laure
Publicado 2023

Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β(A(T87Q))-Globin Gene

por 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
Publicado 2016

An Evaluation of Three Ways of Communicating Carrier Status Results to the Parents of Children in a Neonatal Sickle Cell Screening Programme

por Rémus, Christelle, Stanislas, Aurélie, Bouazza, Naïm, Gauthereau, Valérie, Polak, Michel, Blanche, Stéphane, Niakaté, Assa, Gluckman, Eliane, Tréluyer, Jean-Marc, Munnich, Arnold, Girot, Robert, Cavazzana, Marina
Publicado 2020

Comparing genotoxic signatures in cord blood cells from neonates exposed in utero to zidovudine or tenofovir

por Vivanti, Alexandre, Soheili, Tayebeh S., Cuccuini, Wendy, Luce, Sonia, Mandelbrot, Laurent, Lechenadec, Jerome, Cordier, Anne-Gael, Azria, Elie, Soulier, Jean, Cavazzana, Marina, Blanche, Stéphane, André-Schmutz, Isabelle
Publicado 2015

Bayesian Modeling Immune Reconstitution Apply to CD34+ Selected Stem Cell Transplantation for Severe Combined Immunodeficiency

por Diana, Jean-Sebastien, Bouazza, Naïm, Couzin, Chloe, Castelle, Martin, Magnani, Alessandra, Magrin, Elisa, Rosain, Jeremie, Treluyer, Jean-Marc, Picard, Capucine, Moshous, Despina, Blanche, Stéphane, Neven, Bénédicte, Cavazzana, Marina
Publicado 2022

Two Monogenetic Disorders, Activated PI3-Kinase-δ Syndrome 2 and Smith–Magenis Syndrome, in One Patient: Case Report and a Literature Review of Neurodevelopmental Impact in Primary...

por Moreno-Corona, Nidia, Chentout, Loïc, Poggi, Lucie, Thouenon, Romane, Masson, Cecile, Parisot, Melanie, Mouel, Lou Le, Picard, Capucine, André, Isabelle, Cavazzana, Marina, Perrin, Laurence, Durandy, Anne, Azarnoush, Saba, Kracker, Sven
Publicado 2021

Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID

por Charrier, Sabine, Lagresle-Peyrou, Chantal, Poletti, Valentina, Rothe, Michael, Cédrone, Grégory, Gjata, Bernard, Mavilio, Fulvio, Fischer, Alain, Schambach, Axel, de Villartay, Jean-Pierre, Cavazzana, Marina, Hacein-Bey-Abina, Salima, Galy, Anne
Publicado 2019