Gene Editing of Hematopoietic Stem Cells: Hopes and Hurdles Toward Clinical Translation by Samuele Ferrari, Samuele Ferrari, Valentina Vavassori, Valentina Vavassori, Daniele Canarutto, Daniele Canarutto, Daniele Canarutto, Aurelien Jacob, Aurelien Jacob, Maria Carmina Castiello, Maria Carmina Castiello, Attya Omer Javed, Pietro Genovese, Pietro Genovese, Pietro Genovese, Pietro Genovese

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Paediatric Wolfram syndrome Type 1: should gonadal dysfunction be part of the diagnostic criteria? by Giulio Frontino, Giulio Frontino, Raffaella Di Tonno, Raffaella Di Tonno, Marianna Rita Stancampiano, Marianna Rita Stancampiano, Francesca Arrigoni, Francesca Arrigoni, Andrea Rigamonti, Andrea Rigamonti, Elisa Morotti, Elisa Morotti, Daniele Canarutto, Daniele Canarutto, Riccardo Bonfanti, Riccardo Bonfanti, Riccardo Bonfanti, Gianni Russo, Graziano Barera, Lorenzo Piemonti, Lorenzo Piemonti

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P1405: IN-DEPTH GENOME INTEGRITY EVALUATION AND GMP-COMPLIANT MANUFACTURING OF HDR GENE EDITED CD4+ T CELLS FOR THE TREATMENT OF HYPER IGM 1 by Daniele Canarutto, Claudia Asperti, Valentina Vavassori, Simona Porcellini, Elisabetta Rovelli, Marianna Paulis, Samuele Ferrari, Tiziana Plati, Lucia Sergi Sergi, Anna Villa, Marina Radrizzani, Luigi Naldini

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Case Report: Consistent disease manifestations with a staggered time course in two identical twins affected by adenosine deaminase 2 deficiency by Federica Barzaghi, Maria Pia Cicalese, Maria Pia Cicalese, Maria Pia Cicalese, Matteo Zoccolillo, Immacolata Brigida, Matteo Barcella, Matteo Barcella, Ivan Merelli, Claudia Sartirana, Monica Zanussi, Valeria Calbi, Maria Ester Bernardo, Maria Ester Bernardo, Maria Ester Bernardo, Francesca Tucci, Maddalena Migliavacca, Fabio Giglio, Matteo Doglio, Daniele Canarutto, Daniele Canarutto, Daniele Canarutto, Francesca Ferrua, Giulia Consiglieri, Giulia Consiglieri, Giulia Prunotto, Giulia Prunotto, Francesco Saettini, Sonia Bonanomi, Patrizia Rovere-Querini, Patrizia Rovere-Querini, Giulia Di Colo, Giulia Di Colo, Tatiana Jofra, Georgia Fousteri, Federica Penco, Marco Gattorno, Michael S. Hershfield, Lucia Bongiovanni, Maurilio Ponzoni, Maurilio Ponzoni, Sarah Marktel, Raffaella Milani, Jacopo Peccatori, Fabio Ciceri, Fabio Ciceri, Alessandra Mortellaro, Alessandro Aiuti, Alessandro Aiuti, Alessandro Aiuti

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P1373: UNCOVERING UPSIDES AND PITFALLS OF BASE AND PRIME EDITING IN HEMATOPOIETIC STEM CELLS by Martina Fiumara, Samuele Ferrari, Attya Omer-Javed, Stefano Beretta, Luisa Albano, Daniele Canarutto, Angelica Varesi, Chiara Gaddoni, Chiara Brombin, Federica Cugnata, Erika Zonari, Matteo Maria Naldini, Matteo Barcella, Bernhard Gentner, Ivan Merelli, Luigi Naldini

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Scalable GMP-compliant gene correction of CD4+ T cells with IDLV template functionally validated in vitro and in vivo by Claudia Asperti, Daniele Canarutto, Simona Porcellini, Francesca Sanvito, Francesca Cecere, Valentina Vavassori, Samuele Ferrari, Elisabetta Rovelli, Luisa Albano, Aurelien Jacob, Lucia Sergi Sergi, Elisa Montaldo, Francesca Ferrua, Luis Ignacio González-Granado, Vassilios Lougaris, Raffaele Badolato, Andrea Finocchi, Anna Villa, Marina Radrizzani, Luigi Naldini

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Peripheral blood stem and progenitor cell collection in pediatric candidates for ex vivo gene therapy: a 10-year series by Daniele Canarutto, Francesca Tucci, Salvatore Gattillo, Matilde Zambelli, Valeria Calbi, Bernhard Gentner, Francesca Ferrua, Sarah Marktel, Maddalena Migliavacca, Federica Barzaghi, Giulia Consiglieri, Vera Gallo, Francesca Fumagalli, Paola Massariello, Cristina Parisi, Gianluca Viarengo, Elena Albertazzi, Paolo Silvani, Raffaella Milani, Luca Santoleri, Fabio Ciceri, Maria Pia Cicalese, Maria Ester Bernardo, Alessandro Aiuti

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Outcome of BCG Vaccination in ADA-SCID Patients: A 12-Patient Series by Daniele Canarutto, Chiara Oltolini, Federica Barzaghi, Valeria Calbi, Maddalena Migliavacca, Francesca Tucci, Vera Gallo, Giulia Consiglieri, Francesca Ferrua, Salvatore Recupero, Maria Celia Cervi, Hamoud Al-Mousa, Anna Pituch-Noworolska, Chiara Tassan Din, Paolo Scarpellini, Paolo Silvani, Claudia Fossati, Miriam Casiraghi, Daniela Maria Cirillo, Antonella Castagna, Maria Ester Bernardo, Alessandro Aiuti, Maria Pia Cicalese

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