Dandan Wang, MD, MS^1,2, Fangfei Zhang^3,4*, Anthony Veltri, PhD^5*, John Anto Pulikkan, PhD^6,7, Sridhar Rao, MD, PhD^1,8,9 and Subramaniam Malarkannan, PhD^1,10,11,12

¹Versiti Blood Research Institute, Milwaukee, WI
²Medical College of Wisconsin, Milwaukee, WI
³Medical College of Wisconsin, Milwaukee
⁴Versiti Blood Research Institute, Milwaukee, WI, Milwaukee
⁵Versiti Blood Research Institute, Milwaukee, WI, Milwaukee, WI
⁶Department of Cell Biology, Neurobiology and Anatomy, Medical College of Wisconsin, Milwaukee, WI
⁷Blood Research Institute Versiti, Milwaukee, WI
⁸Department of Pediatrics, Division of Hematology, Oncology, and Transplantation, Medical College of Wisconsin, Milwaukee, WI
⁹Cell Biology, Neurobiology, and Anatomy, Medical College of Wisconsin, Milwaukee, WI
¹⁰Blood Research Institute, Milwaukee, WI
¹¹Microbiology & Immunology, Medical College of Wisconsin, Milwaukee, WI
¹²Department of Medicine, Medical College of Wisconsin, Milwaukee, WI

Melissa Mavers, MD, PhD^1*, Derek Hollingsworth, MS^2*, Reyka G. Jayasinghe, PhD^3*, Chaiyaporn Boonchalermvichian, MD, PhD⁴, Biki Gupta, PhD^4*, Hao Yan, PhD^4*, Jeanette Baker, PhD^4*, Beruh Dejene^5*, Kenneth I. Weinberg, MD^5* and Robert S. Negrin, MD⁴

¹Department of Pediatrics, Division of Hematology and Oncology, Washington University School of Medicine, St. Louis, MO
²insitro, South San Francisco
³Department of Internal Medicine, Division of Oncology, Washington University School of Medicine, St. Louis, MO
⁴Department of Medicine, Division of Blood and Marrow Transplantation and Cellular Therapy, Stanford University School of Medicine, Stanford, CA
⁵Department of Pediatrics, Division of Hematology, Oncology, Stem Cell Transplantation and Regenerative Medicine, Stanford University School of Medicine, Stanford, CA

Francesco Saettini, MD^1*, Fabiola Guerra^2,3,4*, Mario Mauri, PhD^5*, Grazia Fazio, PhD^6*, Cristina Bugarin, MSc^7*, Manuel Quadri^2*, Stefano Rebellato^4,7*, Clizia Chinello^4*, Lisa Pagani^4*, Federica Malighetti, MS^5*, Luis González Gutiérrez-Solana^8*, Vanna Denti^4*, Fatemeh Emam Mousavi^9,10*, Miquell Raspall-Chaure^11*, Martin-Nalda Andrea^12*, Estibaliz Barredo^13*, David Adams^14*, O'Leary Melanie^15*, Precilla D’Souza^14*, Ellen Macnamara^14*, Sergio Rosenzweig^16*, Hye Sun Kuehn^16*, Jennifer Stoddard^16*, Heather C Mefford^17*, Giorgia Mandrile^18*, Lisa Pavinato^19*, Alfredo Brusco^19,20*, Patricia Valentina Velez Santamaria^21*, Aurora Pujol^21*, Vincent Michaud^22*, Agathe Roubertie^23*, Zoe Nelson^24*, Margaret P Adam^25*, Bernice Lo^26*, Holm Uhlig^27,28,29*, Claire G Salter^30,31*, Emma Baple^31,32*, Andrew H Crosby^31*, Sanil Bhatia, PhD^33*, Fulvio Magni^4*, Giuseppe Paglia^4*, Giovanni Cazzaniga, PhD^4,34, Rocco Piazza, MD, PhD^35*, Andrea Biondi, MD^3,4,36 and Matteo Barberis^10,37,38*

¹Centro Tettamanti, IRCCS San Gerardo dei Tintori, MERATE, Italy
²Centro Tettamanti, Fondazione IRCCS San Gerardo dei Tintori, Monza, ITA
³Pediatria, Fondazione IRCCS San Gerardo dei Tintori, Monza, Italy
⁴Dipartimento di Medicina e Chirurgia, Università degli Studi Milano-Bicocca, Monza, Italy
⁵Department of Medicine and Surgery, University of Milano-Bicocca, Monza, ITA
⁶Tettamanti Research Center, Monza, MB, ITA
⁷Tettamanti Center, Fondazione IRCCS San Gerardo dei Tintori, Monza, Italy
⁸Hospital Infantil Niño Jesús, Madrid, España or Infant Jesus Children’s Hospital, Madrid, Spain
⁹Molecular Systems Biology, School of Biosciences, Faculty of Health and Medical Sciences, University of Surrey, Guildford, United Kingdom
¹⁰Centre for Mathematical and Computational Biology, CMCB, University of Surrey, Guildford, United Kingdom
¹¹Department of Paediatric Neurology, Hospital Universitari Vall d'Hebron, Barcelona, Spain
¹²8. Pediatric Infectious Diseases and Immunodeficiencies Unit, Hospital Universitari Vall d'Hebron, Institut de Recerca Vall d'Hebron, Universitat Autònoma de Barcelona, Barcelona, Spain
¹³Neuropediatric Department, Hospital Universitario Gregorio Marañón, Madrid, Spain
¹⁴NIH Undiagnosed Diseases Program, NIH, Bethesda
¹⁵11. Center for Mendelian Genomics, Program in Medical and Population Genetics, Broad Institute of MIT and Harvard, Cambridge
¹⁶Immunology Service, DLM, NIH Clinical Center, Bethesda
¹⁷Division of Genetic Medicine, Department of Paediatrics, University of Washington, Seattle
¹⁸14. Genetic Unit and Thalassemia Center, San Luigi Gonzaga University Hospital, Orbassano, Italy
¹⁹Department of Medical Sciences, University of Turin, Torino, Italy
²⁰Medical Genetics Unit, Città della Salute e della Scienza University Hospital, Torino, Italy
²¹Neurometabolic Diseases Laboratory, Bellvitge Biomedical Research Institute (IDIBELL), L'Hospitalet de Llobregat, Barcelona, Spain
²²18. Molecular Genetics Laboratory, Bordeaux University Hospital, Bordeaux, Aquitaine, France. INSERM U1211, Rare Diseases Laboratory: Genetics and Metabolism, University of Bordeaux, Talence, FRA
²³19. Département de Neuropédiatrie, CIC, CHU de Montpellier, INM, Univ Montpellier, INSERM U 1298, Montpellier, France
²⁴Vascular Anomalies Program, Seattle Children’s Hospital, Seattle
²⁵Genetic Medicine, Department of Pediatrics, University of Washington, Seattle
²⁶College of Health and Life Sciences, Hamad Bin Khalifa University, Doha, Qatar
²⁷Department of Paediatrics, University of Oxford, Oxford, United Kingdom
²⁸Oxford NIHR Biomedical Research Centre, Oxford, United Kingdom
²⁹University of Oxford, Oxford, GBR
³⁰Wessex Clinical Genetics Service, Princess Anne Hospital, Southampton, United Kingdom
³¹RILD Wellcome Wolfson Centre, University of Exeter Medical School, Exeter, United Kingdom
³²Peninsula Clinical Genetics Service, Royal Devon and Exeter Hospital, Exeter, United Kingdom
³³Department of Pediatric Oncology, Hematology and Clinical Immunology, Medical faculty, Heinrich Heine University Düsseldorf, Duesseldorf, Germany
³⁴Centro Tettamanti, Fondazione IRCCS San Gerardo dei Tintori, Monza, Italy
³⁵Department of Medicine and Surgery, University of Milano-Bicocca, Monza, Italy
³⁶Centro Tettamanti, Fondazione IRCCS San Gerardo dei Tintori,, Monza, Italy
³⁷4. Molecular Systems Biology, School of Biosciences, Faculty of Health and Medical Sciences, University of Surrey, Guildford, United Kingdom
³⁸Synthetic Systems Biology and Nuclear Organization, Swammerdam Institute for Life Sciences, University of Amsterdam, Amsterdam, Netherlands

Carlos Bravo-Perez, MD^1,2*, Carmelo Gurnari, MD^1,3, Luca Guarnera, MD^1*, Naomi Kawashima, MD, PhD^1*, Serhan Unlu, MD^1*, Aashray Mandala, BS^1*, Mark Orland, MD¹, Olisaemeka Ogbue¹, Nakisha D Williams, MBBS^1*, Arooj Ahmed, MD^1*, Yasuo Kubota, MD, PhD^4*, Arda Durmaz^1*, Valeria Visconte, PhD¹ and Jaroslaw P. Maciejewski, M.D., Ph.D., FACP¹

¹Department of Translational Hematology and Oncology Research, Taussig Cancer Institute, Cleveland Clinic, Cleveland, OH
²Department of Hematology and Medical Oncology, Hospital Universitario Morales Meseguer, University of Murcia, IMIB-Pascual Parrilla, CIBERER - Instituto de Salud Carlos III, Murcia, Spain
³Department of Biomedicine and Prevention, University of Rome Tor Vergata, Rome, Italy
⁴Department of Translational Hematology and Oncology Research, Taussig Cancer Institute, Cleveland Clinic, Beachwood, OH

Yongguang Zhang, PhD^1*, Mei Yu, PhD^1*, Yongwei Zheng, PhD^1*, Yuhong Chen^1*, Jesus Izaguirre-Carbonell^2*, Nan Zhu, PhD³, Renren Wen, PhD^1,4 and Demin Wang, PhD^4,5

¹Versiti Blood Research Institute, Milwaukee, WI
²Blood Research Institute, Versiti Inc., Milwaukee, WI
³Versiti Blood Research Institute Milwaukee, Milwaukee, WI
⁴Department of Microbiology and Immunology, Medical College of Wisconsin, Milwaukee, WI
⁵The Blood Research Institute, Milwaukee, WI

Benjamin R Babcock, BS¹, Astrid Kosters^1*, Nadia R Roan^2,3*, Sulggi Lee^4,5* and Eliver E.B. Ghosn, PhD^6*

¹Department of Medicine, Division of Immunology, Lowance Center for Human Immunology, Emory University, Atlanta, GA
²Gladstone Institutes, San Francisco
³Department of Urology, University of California San Francisco, San Francisco
⁴Zuckerberg San Francisco General Hospital, San Francisco
⁵Department of Medicine, University of California San Francisco, San Francisco
⁶Department of Medicine, Division of Immunology, Lowance Center for Human Immunology, Emory Vaccine Center, Emory National Primate Research Center, Emory University, Atlanta, GA