Human mutations affect the epigenetic/bookmarking function of HNF1B

por Lerner, Jonathan, Bagattin, Alessia, Verdeguer, Francisco, Makinistoglu, Munevver P., Garbay, Serge, Felix, Tristan, Heidet, Laurence, Pontoglio, Marco
Publicado 2016

GATA Factor-Mediated Gene Regulation in Human Erythropoiesis

por Romano, Oriana, Petiti, Luca, Felix, Tristan, Meneghini, Vasco, Portafax, Michel, Antoniani, Chiara, Amendola, Mario, Bicciato, Silvio, Peano, Clelia, Miccio, Annarita
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

A Nonintegrative Lentiviral Vector-Based Vaccine Provides Long-Term Sterile Protection against Malaria

por Coutant, Frédéric, Sanchez David, Raul Yusef, Félix, Tristan, Boulay, Aude, Caleechurn, Laxmee, Souque, Philippe, Thouvenot, Catherine, Bourgouin, Catherine, Beignon, Anne-Sophie, Charneau, Pierre
Publicado 2012

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

por 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
Publicado 2018

Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype

por Weber, Leslie, Frati, Giacomo, Felix, Tristan, Hardouin, Giulia, Casini, Antonio, Wollenschlaeger, Clara, Meneghini, Vasco, Masson, Cecile, De Cian, Anne, Chalumeau, Anne, Mavilio, Fulvio, Amendola, Mario, Andre-Schmutz, Isabelle, Cereseto, Anna, El Nemer, Wassim, Concordet, Jean-Paul, Giovannangeli, Carine, Cavazzana, Marina, Miccio, Annarita
Publicado 2020

Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem cells in sickle cell disease patients after exchange transfusion

por Lagresle-Peyrou, Chantal, Lefrère, François, Magrin, Elisa, Ribeil, Jean-Antoine, Romano, Oriana, Weber, Leslie, Magnani, Alessandra, Sadek, Hanem, Plantier, Clémence, Gabrion, Aurélie, Ternaux, Brigitte, Félix, Tristan, Couzin, Chloé, Stanislas, Aurélie, Tréluyer, Jean-Marc, Lamhaut, Lionel, Joseph, Laure, Delville, Marianne, Miccio, Annarita, André-Schmutz, Isabelle, Cavazzana, Marina
Publicado 2018

Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression

por Antoniou, Panagiotis, Hardouin, Giulia, Martinucci, Pierre, Frati, Giacomo, Felix, Tristan, Chalumeau, Anne, Fontana, Letizia, Martin, Jeanne, Masson, Cecile, Brusson, Megane, Maule, Giulia, Rosello, Marion, Giovannangeli, Carine, Abramowski, Vincent, de Villartay, Jean-Pierre, Concordet, Jean-Paul, Del Bene, Filippo, El Nemer, Wassim, Amendola, Mario, Cavazzana, Marina, Cereseto, Anna, Romano, Oriana, Miccio, Annarita
Publicado 2022