Martina Fiumara, PhD1,2*, Raffaella Molteni, PhD2,3,4*, Corrado Campochiaro, MD3,5*, Roberta Alfieri, PhD6*, Guido Pacini, PhD1*, Alessandro Tomelleri, MD3,5*, Elisa Diral, MD7*, Angelica Varesi, MSc1,8,9*, Alessandra Weber, MD1,3*, Pamela Quaranta, PhD1*, Luisa Albano, MSc1*, Chiara Gaddoni, MSc1*, Luca Basso-Ricci, MSc1*, Davide Stefanoni, PhD4*, Gregorio Maria Bergonzi, MD7*, Andrea Annoni, PhD1*, Maddalena Panigada, PhD4*, Eleonora Cantoni, MSc4*, Daniele Canarutto, MD, PhD1,10*, Stephanie Z Xie, PhD8, Angelo D'Alessandro, PhD11, Raffaella Di Micco, PhD1,12*, Alessandro Aiuti, MD, PhD1,3,10*, Fabio Ciceri, MD3,13*, Giacomo De Luca, MD3,5*, Lorenzo Dagna, MD3,5*, Marco Matucci-Cerinic, MD3,5*, Ivan Merelli, PhD1,6*, Simone Cenci, MD3,4, Serena Scala, PhD1*, Giulio Cavalli, MD, PhD3,4,14*, Luigi Naldini, MD, PhD1,3,14 and Samuele Ferrari, PhD1,3,14
1San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy
2co-first author, Milan, Italy
3Vita-Salute San Raffaele University, Milan, Italy
4Division of Genetics and Cell Biology, IRCCS San Raffaele Scientific Institute, Milan, Italy
5Unit of Immunology, Rheumatology, Allergy and Rare diseases, IRCCS San Raffaele Hospital, Milan, Italy
6Institute for Biomedical Technologies, National Research Council, Segrate, Italy
7Unit of Hematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, Milan, Italy
8Princess Margaret Cancer Centre, Toronto, ON, Canada
9University of Toronto, Department of Molecular Genetics, Toronto, Canada
10Pediatric Immunohematology Unit and BMT Program, IRCCS San Raffaele Scientific Institute, Milan, Italy
11Department of Biochemistry and Molecular Genetics, University of Colorado Anschutz Medical Campus, Aurora, CO
1211 Istituto Universitario di Studi Superiori, IUSS, Pavia, Italy
13Hematology and Bone Marrow Transplantation Unit, I.R.C.C.S. San Raffaele Scientific Institute, Milan, Italy
14co-last author, Milan, Italy