Resultados da pesquisa - Marina Cavazzana

Autologous Stem-Cell-Based Gene Therapy for Inherited Disorders: State of the Art and Perspectives Por Frank J. T. Staal, Alessandro Aiuti, Alessandro Aiuti, Marina Cavazzana

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An Evaluation of Three Ways of Communicating Carrier Status Results to the Parents of Children in a Neonatal Sickle Cell Screening Programme Por Christelle Rémus, Christelle Rémus, Christelle Rémus, Aurélie Stanislas, Aurélie Stanislas, Naïm Bouazza, Valérie Gauthereau, Michel Polak, Stéphane Blanche, Assa Niakaté, Eliane Gluckman, Jean-Marc Tréluyer, Arnold Munnich, Robert Girot, Robert Girot, Robert Girot, Marina Cavazzana, Marina Cavazzana

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Development of Lentiviral Vectors Simultaneously Expressing Multiple siRNAs Against CCR5, vif and tat/rev Genes for an HIV-1 Gene Therapy Approach Por Francesca Spanevello, Arianna Calistri, Claudia Del Vecchio, Barbara Mantelli, Chiara Frasson, Giuseppe Basso, Giorgio Palù, Marina Cavazzana, Cristina Parolin

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Bayesian Modeling Immune Reconstitution Apply to CD34+ Selected Stem Cell Transplantation for Severe Combined Immunodeficiency Por Jean-Sebastien Diana, Jean-Sebastien Diana, Naïm Bouazza, Naïm Bouazza, Chloe Couzin, Martin Castelle, Alessandra Magnani, Elisa Magrin, Jeremie Rosain, Jean-Marc Treluyer, Jean-Marc Treluyer, Capucine Picard, Capucine Picard, Despina Moshous, Despina Moshous, Stéphane Blanche, Stéphane Blanche, Bénédicte Neven, Bénédicte Neven, Marina Cavazzana, Marina Cavazzana

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

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Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies Por Mégane Brusson, Anne Chalumeau, Pierre Martinucci, Oriana Romano, Tristan Felix, Valentina Poletti, Samantha Scaramuzza, Sophie Ramadier, Cecile Masson, Giuliana Ferrari, Fulvio Mavilio, Marina Cavazzana, Mario Amendola, Annarita Miccio

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Sustained remission after haploidentical bone marrow transplantation in a child with refractory systemic juvenile idiopathic arthritis Por Guillaume Morelle, Martin Castelle, Graziella Pinto, Sylvain Breton, Matthieu Bendavid, Charlotte Boussard, Richard Mouy, Brigitte Bader-Meunier, Michaela Semeraro, Albert Faye, Marina Cavazzana, Bénédicte Neven, Stéphane Blanche, Pierre Quartier, Despina Moshous

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Limiting Thymic Precursor Supply Increases the Risk of Lymphoid Malignancy in Murine X-Linked Severe Combined Immunodeficiency Por Samantha L. Ginn, Claus V. Hallwirth, Sophia H.Y. Liao, Erdahl T. Teber, Jonathan W. Arthur, Jianmin Wu, Hong Ching Lee, Szun S. Tay, Min Hu, Roger R. Reddel, Matthew P. McCormack, Adrian J. Thrasher, Marina Cavazzana, Stephen I. Alexander, Ian E. Alexander

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