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