Francesca Ferrua, MD^1,2,3*, Maria Pia Cicalese, MD, PhD^1,3*, Stefania Galimberti, PhD^4*, Samantha Scaramuzza, PhD^1*, Stefania Giannelli, PhD^1*, Roberta Pajno, MD^2,3*, Francesca Dionisio, PhD^1*, Luca Biasco, PhD^1*, Maria Carmina Castiello, PhD^1*, Miriam Casiraghi, RN^1,3*, Marcella Facchini, PhD^1*, Andrea Finocchi, MD^5*, Ayse Metin, MD, PhD^6*, Jordan S. Orange, MD, PhD^7*, Michael H. Albert, MD^8*, Carmen Petrescu, MD^9*, Marita Bosticardo, PhD^1*, Anna Villa, MD^1,10*, Chris Dott, PhD^11*, Koen van Rossem, MD, MSc, PhD^12*, Maria Grazia Valsecchi, PhD⁴, Fabio Ciceri, MD^13*, Maria Grazia Roncarolo, MD^1,2,14, Luigi Naldini, MD, PhD^1,2* and Alessandro Aiuti, MD, PhD^1,2,3

¹San Raffaele Telethon Institute for Gene Therapy (TIGET), San Raffaele Scientific Institute, Milan, Italy
²Vita-Salute San Raffaele University, Milan, Italy
³Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Milan, Italy
⁴Center of Biostatistics for Clinical Epidemiology, Department of Health Sciences, University of Milano-Bicocca, Monza, Italy
⁵DPUO, University Department of Pediatrics, Bambino Gesù Children’s Hospital and Tor Vergata University, School of Medicine, Rome, Italy
⁶Pediatric Immunology and Allergy Department, Ankara Children's Hematology Oncology Training and Research Hospital, Ankara, Turkey
⁷Center for Human Immunobiology, Texas Children's Hospital and Baylor College of Medicine, Houston, TX
⁸Dr von Hauner Children's Hospital, Ludwig Maximillian’s University, Munich, Germany
⁹Department of Public Health, School of Medicine, V. Babeş University of Medicine & Pharmacy, Timişoara, Romania
¹⁰Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy
¹¹CSD Pharma Consulting Ltd., London, United Kingdom
¹²Rare Diseases Unit, GlaxoSmithKline, Brentford, United Kingdom
¹³Hematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Milan, Italy
¹⁴Division of Stem Cell Transplantation and Regenerative Medicine and iSCBRM, Department of Pediatrics, Stanford University, Stanford, CA

Julia I Chu, MD, MPH^1,2, Lauren A Henderson, MD^3*, Myriam Armant, PhD^2*, Frances Male, BA^4*, Colleen H Dansereau, MSN, RN^5*, Brenda MacKinnon, RN^6*, Christopher J Burke^1*, Matthew E Cavanaugh^1*, Wendy B. London, PhD^1,2*, Isil B Barlan, MD^7*, Ahmet Özen, MD^7*, Safa Baris, MD^7*, Elif Karakoc-Aydiner, MD^7*, Ayça Kiykim, MD^7*, Imelda Celine Hanson, MD^8*, Jenny M. Despotovic, DO, MS⁹, Akihiko Saitoh, MD, PhD^10*, Takayuki Takachi, MD, PhD^10*, Kohsuke Imai, MD, PhD^11*, Alejandra King, MD^12*, Silvia Arredondo, MD^13*, Jerome Ritz, MD¹⁴, Frederic D Bushman, PhD^4*, Anne Galy, PharmD, PhD¹⁵, Luigi Daniele Notarangelo, MD^3*, David A. Williams, MD^1,2 and Sung-Yun Pai, MD^1,2

¹Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA
²Division of Hematology-Oncology, Boston Children's Hospital, Boston, MA
³Division of Immunology, Boston Children's Hospital, Boston, MA
⁴Department of Microbiology, University of Pennsylvania School of Medicine, Philadelphia, PA
⁵Division of Pediatric Hematology, Boston Children's Hospital, Boston, MA
⁶Division of Pediatric Hematology, Boston Children’s Hospital, Boston, MA
⁷Division of Pediatric Allergy/Immunology, Marmara University, Istanbul, Turkey
⁸Division of Pediatric Immunology, Allergy and Rheumatology, Texas Children's Hospital, Houston, TX
⁹Texas Children's Hematology Center, Baylor College of Medicine, Houston, TX
¹⁰Department of Pediatrics, Niigata University Graduate School of Medical and Dental Sciences, Niigata, Japan
¹¹Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University, Tokyo, Japan
¹²Hospital Luis Calvo Mackenna, Santiago, Chile
¹³Pediatric Service, Hospital Regional Talca, Talca, Chile
¹⁴Division of Hematologic Malignancies, Dana-Farber Cancer Institute, Boston, MA
¹⁵Genethon, Evry, France

Suk See De Ravin, MD PhD^1*, Xiaolin Wu^2*, Narda Theobald^3*, Janet S Lee^4*, John Gray, Ph.D.^5*, Imelda Celine Hanson, MD^6*, Luigi Notarangelo, MD PhD^7*, Brian P. Sorrentino, MD^8,9 and Harry L. Malech, MD¹⁰

¹LHD, National Institutes of Health, Bethesda
²Cancer Reserach Technology Program, Leidos Biomedical Reserach, Inc., Frederick
³LHD, NIAID, NIH, Bethesda, MD
⁴NIH, Bethesda
⁵Research & Developement, Audentes Therapeutics, San Francisco, CA
⁶Division of Pediatric Immunology, Allergy and Rheumatology, Texas Children's Hospital, Houston, TX
⁷Harvard Medical School, Boston
⁸Division of Experimental Hematology, St. Jude Children's Research Hospital, Memphis, TN
⁹Hematology, St. Jude Children's Research Hospital, Memphis, TN
¹⁰Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, Bethesda, MD

Olivier Humbert, PhD^1*, Christopher R Burtner, PhD^1*, Patricia O'Donnell^2*, Daniel R Humphrys, B.S.^1*, Nicholas Hubbard^3*, Frieda Chan, PhD^1*, Jennifer E Adair, PhD^1,4*, Grant D. Trobridge, PhD^5*, Troy Torgerson, MD, PhD^3,4*, Andrew M. Scharenberg, MD^4*, David J. Rawlings, MD^3,4*, Peter Felsburg^2* and Hans-Peter Kiem, MD^1,4

¹Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA
²University of Pennsylvania, philadelphia, PA
³Seattle Children's Research Institute, Seattle, WA
⁴University of Washington, Seattle, WA
⁵Washington State University, Pullman, WA

Giacomo Oliveira, PhD^1,2*, Eliana Ruggiero, PhD^3,4*, Maria Teresa Lupo Stanghellini, MD^5*, Nicoletta Cieri, MD, PhD^3*, Mattia D'Agostino, MD^3*, Raffaele Fronza, PhD^4*, Christina Lulay^4*, Francesca Dionisio, PhD^6*, Sara Mastaglio, MD^3,5*, Raffaella Greco, MD^7*, Jacopo Peccatori, MD^5*, Alessandro Aiuti, MD⁸, Luca Biasco, PhD^6*, Attilio Bondanza, MD, PhD⁹, Antonio Lambiase, MD^10*, Catia Traversari, PhD^10*, Luca Vago, MD, PhD^7,11, Christof von Kalle, MD^4*, Manfred Schmidt, PhD^4*, Claudio Bordignon, MD^1,10, Fabio Ciceri, MD^12* and Chiara Bonini, MD¹³

¹Vita-Salute San Raffaele University, Milan, Italy
²Experimental Hematology Unit, San Raffaele Scientific Institute, Milano, Italy
³Experimental Hematology Unit, San Raffaele Scientific Institute, Milan, Italy
⁴Department of Translational Oncology, National Center for Tumor Diseases and German Cancer Research Center, Heidelberg, Germany
⁵Hematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Milan, Italy
⁶San Raffaele Telethon Institute for Gene Therapy (TIGET), San Raffaele Scientific Institute, Milan, Italy
⁷Unit of Hematology and Bone Marrow Transplantation, San Raffaele Scientific Institute, Milano, Italy
⁸Telethon Institute for Gene Therapy (HSR-TIGET), San Raffaele Scientific Institute, Milan, Italy
⁹Innovative Immunotherapies Unit, San Raffaele Hospital Scientific Institute, Milan, Italy
¹⁰MolMed S.p.A., Milan, Italy
¹¹Unit of Immunogenetics, Leukemia Genomics and Immunobiology, San Raffaele Scientific Institute, Milano, Italy
¹²Hematology and Bone Marrow Transplantation Unit, San Raffaele Hospital Scientific Institute, Milan, Italy
¹³Experimental Hematology Unit, San Raffaele Hospital Scientific Institute, Milan, Italy

Lung-Ji Chang, PhD^1,2, Lujia Dong, M.D.³, Jun Zhu, MD⁴, Zhitao Ying, MD^4*, Hao-Hsian Kuo, MSc^1,3*, Yuchen Liu, MSc^1,3*, Yu-Qin Song, M.D.^4*, Xiao-Pei Wang, M.D.^4*, Yong-Qian Jia, M.D.^5*, Ting Niu, MD, PhD⁵, Ting Liu, MD, PhD⁵, Ling Pan, M.D.^5*, Zhuo-Gang Liu, M.D.^6*, Tong Li, M.D.^6*, Ying-Chun Li, M.D.^7*, Kun Yao, MD^7*, Xiaoyan Ke, MD, PhD^7*, Hongmei Jing, M.D.^8* and Fang Bao, M.D.^8*

¹Shenzhen Genoimmune Medical Institute (GIMI), Shenzhen, China
²Department of Molecular Genetics and Microbiology, University of Florida, Gainesville, FL
³America Yuva Biomed, Beijing, China
⁴Peking University Cancer Hospital, Beijing, China
⁵Department of Hematology, West China Hospital, Sichuan University, Chengdu, China
⁶Department of Hematology, Sheng-Jing Hospital of China Medical University, Shen-Yang, China
⁷Department of Hematology, Third Affiliated Hospital of Peking University, Beijing, China
⁸Department of Hematology, Peking University Third Hospital, Beijing, China