Liying Guan, PhD^1*, Deepak Voora, MD^2*, Rachel A Myers, PhD^2* and Angara Koneti Rao, MBBS¹

¹Sol Sherry Thrombosis Research Center, Lewis Katz School of Medicine at Temple University, Philadelphia, PA
²Center for Applied Genomics & Precision Medicine, Duke University School of Medicine, Durham, NC

Michael J. Peters^1*, Anna Deck^1*, April Hu, MD, PhD^2*, Yuko Fujiwara, PhD^1*, Jinkuk Kim, PhD^2*, Aubrie Soucy, MA^2*, Kevin Zhang^1*, Julianna Kenny^1*, Linda Y. Lin^1*, Alyssa L. Kennedy, MD, PhD¹, Caitlin Bowers^1*, Chad E. Harris, MS^1*, Chi Yuan Zhang, PhD^1*, Karthik Ponnienselvan^3*, Pengpeng Liu, PhD^3*, Kevin Luk, PhD^3*, Davide Seruggia, PhD^4,5*, R. Coleman Lindsley⁶, John P. Manis, MD^1*, Christian Brendel, PhD^1*, Scot A. Wolfe, PhD^3*, Stuart H. Orkin, MD¹, Timothy Yu, MD, PhD^2*, Akiko Shimamura¹ and Daniel E. Bauer, MD, PhD¹

¹Division of Hematology/Oncology, Boston Children's Hospital, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA
²Division of Genetics and Genomics, Boston Children’s Hospital, Harvard Medical School, Boston, MA
³Department of Molecular, Cell and Cancer Biology, Li Weibo Institute for Rare Diseases Research, University of Massachusetts Medical School, Worcester, MA
⁴CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences, Vienna, Austria
⁵St. Anna Children’s Cancer Research Institute (CCRI), Vienna, Austria
⁶Department of Medical Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA

Yuefeng Tang, PhD¹, Mushran Kahn, B.S^1*, Julien Papoin, MS^1*, Hongxia Yan, MS^2*, Anupama Narla, MD³, James Palis, MD⁴, Laurie A. Steiner, MD⁵, Patrick G. Gallagher, MD^6,7,8, Jeffrey M Lipton, MD, PhD⁹, Mohandas Narla, DSc² and Lionel Blanc, PhD¹

¹The Feinstein Institutes for Medical Research, Manhasset, NY
²Laboratory of Red Cell Physiology, New York Blood Center, New York, NY
³Division of Hematology/Oncology, Department of Pediatrics, Stanford University School of Medicine, Hillsborough, CA
⁴Department of Pathology and Laboratory Medicine, University of Rochester School of Medicine and Dentistry, Rochester, NY
⁵Department of Pediatrics, University of Rochester, Rochester, NY
⁶Departments of Pediatrics, Pathology, and Genetics, Yale School of Medicine, New Haven, CT
⁷Yale School of Medicine, New Haven, CT, USA, New Haven, NY
⁸Pediatrics, Pathology, Genetics, Yale School of Medicine, Hamden, CT
⁹Hematology/Oncology and Stem Cell Transplantation, Cohen Children's Medical Center of NY, New Hyde Park, NY

Arman M Niknafs, BSc^1,2*, Sharon A. Savage, MD³, Marena Rebekka Niewisch, MD^4,5* and Neelam Giri, MD⁶

¹School of Medicine, University of Rochester School of Medicine and Dentistry, Rochester, NY
²Clinical Genetics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, National Institutes of Health, Rochester, NY
³Clinical Genetics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, Rockville, MD
⁴Clinical Genetics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, National Institutes of Health, Rockville, MD
⁵Department of Pediatric Hematology and Oncology, Hannover Medical School, Hannover, Germany
⁶Clinical Genetics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, Bethesda, MD

Ashley Galvin, BS^1*, Chris R. Reilly, MD², Maggie Malsch, MSN, RN^3*, Sara Loveless, BSN^4*, Joseph H. Antin, MD⁵, Stella M Davies, MBBS, PhD, MRCP⁶, Courtney D. DiNardo, MD, MSCE⁷, Adam Lane, PhD^8*, Coleman Lindsley, MD, PhD^2*, Taizo A. Nakano, MD⁹, Richard Cooper^10*, Katherine Coyne^11*, Claire Dusa^10*, Margret Joos^12*, Mark D. Fleming, MD, DPhil¹³, Kasiani C. Myers, MD¹⁴ and Akiko Shimamura, MD, PhD^15*

¹Boston Children's Hospital, Boston, MA
²Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA
³Boston Children's Hospital, East Boston, MA
⁴Cincinnati Children’s Hospital Medical Center, Mason, OH
⁵Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA
⁶Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH
⁷Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX
⁸Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
⁹Center for Cancer and Blood Disorders, Children's Hospital Colorado, Denver, CO
¹⁰Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
¹¹Department of Hematology/Oncology, Boston Children's Hospital, Boston, MA
¹²Cancer and Blood Disorders Center, Dana-Farber/Boston Children’s, Boston, MA
¹³Department of Pathology, Boston Children's Hospital and Harvard Medical School, Boston, MA
¹⁴Department of Pediatrics, Division of Bone Marrow Transplantation and Immune Deficieny, Cincinnati Children's Hospital, Cincinnati, OH
¹⁵Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Boston, MA

Robert Meyer^1*, Matthias Begemann^1*, Marcus Jakob^2*, Claudia Khurana, MD^3*, Daniela Dey^1*, Tarek Hanafee Alali, MD^2*, Heinz Gabriel^4*, Kim Kricheldorf^5,6*, Margherita Vieri, PhD^5,6*, Susanne Isfort^5,6*, Martin Kirschner, MD^5,6*, Jens Panse, MD^5,6, Udo Kontny, MD^7*, Selim Corbacioglu, MD², Tim H. Brümmendorf, MD^5,6, Ingo Kurth, MD^1,6* and Fabian Beier, MD^5,6*

¹Institute for Human Genetics and Genomic Medicine, Medical Faculty, RWTH Aachen University, Aachen, Germany
²Department of Pediatric Hematology, Oncology and Stem Cell Transplantation, University Hospital of Regensburg, Regensburg, Germany
³Department for Pediatric Medicine, Evangelisches Klinikum Bethel, Bielefeld, Germany, Bielefeld, Germany
⁴Center for Human Genetics Tübingen, Tübingen, Germany, Tübingen, Germany
⁵Department of Hematology, Oncology, Hemostaseology and Stem Cell Transplantation, Medical Faculty, RWTH Aachen University, Aachen, Germany
⁶Center for Integrated Oncology Aachen Bonn Cologne Düsseldorf (CIO ABCD), Aachen, Germany
⁷Division of Pediatric Hematology, Oncology and Stem Cell Transplantation, Medical Faculty, Germany, RWTH Aachen University, Aachen, Germany

Hannah A. Raj, BS^1*, Tsung-Po Lai, PhD^2*, Marena R. Niewisch, MD^1,3*, Youjin Wang, PhD, BPharm^1*, Stephen R. Spellman^4*, Abraham Aviv, MD^2*, Shahinaz M. Gadalla, MD, PhD¹ and Sharon A. Savage, MD¹

¹Clinical Genetics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, Rockville, MD
²Center of Human Development and Aging, Rutgers University of New Jersey, New Jersey Medical School, Newark, NJ
³Department of Pediatric Hematology and Oncology, Medical School Hannover, Hannover, Germany
⁴CIBMTR® (Center for International Blood and Marrow Transplant Research), National Marrow Donor Program/Be The Match, Minneapolis, MN

Richa Sharma, MD¹, Senthil Bhoopalan, MBBS, PhD^1*, Lei Han, PhD^1*, Yu Yao, MD^2*, Kalin Mayberry, BS^2*, Baranda S Hansen^3*, Shondra Miller^3*, Claudia Khurana, MD^4*, Miriam Erlacher, MD, PhD^5,6* and Marcin W. Wlodarski, MD, PhD⁷

¹Department of Hematology, St Jude Children's Research Hospital, Memphis, TN
²Department of Hematology, St Jude Children’s Research Hospital, Memphis, TN
³Center for Advanced Genome Engineering (CAGE), St Jude Children's Research Hospital, Memphis, TN
⁴Universität Bielefeld, Bielefeld, Germany
⁵German Cancer Consortium (DKTK), Heidelberg, Germany
⁶Division of Pediatric Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, University of Freiburg, Freiburg, Germany
⁷Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN

Samuele Roncareggi^1*, Katia Girardi, MD^2*, Francesca Fioredda, MD^3*, Francesca Conti^4*, Erika Borlenghi, M.D.^5*, Concetta Micalizzi^6*, Gaia Mancuso^7*, Fabiola Guerra, MD, PhD^8*, Emilia Cirillo^9*, Antonio Marzollo, MD^10*, Piero Farruggia, MD^11*, Daniela Onofrillo^12*, Riccardo Masetti, Prof., MD, PhD^13*, Raffaele Badolato, MD, PhD^14*, Luca Arcuri^15*, Tiziana Angela Coliva, MD^16*, Francesca Vendemini, MD^17*, Claudio Pignata^18*, Sonia Bonanomi^19*, Andrea Biondi, MD²⁰ and Francesco Saettini, MD^21*

¹Milano Bicocca University, Monza, Italy
²Department of Hematology/Oncology, Cell and Gene Therapy, IRCCS Bambino Gesù Children’s Hospital, Rome, Italy
³Hematology Unit, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS)-Istituto Giannina Gaslini, Genova, Italy
⁴Pediatric Unit, IRCCS Azienda Ospedaliero-Universitaria di Bologna, Bologna, ITA
⁵Department of Hematology, ASST Spedali Civili di Brescia, Brescia, Italy
⁶Haematology Unit, IRCCS Istituto Giannina Gaslini, Genoa, Italy
⁷Unit of Immunology, Rheumatology, Allergy and Rare Diseases (UnIRAR), IRCCS San Raffaele Scientific Institute, Milan, Italy., Milan, Italy
⁸University of Milano Bicocca, Monza, Italy, Monza, AL, Italy
⁹"federico II" University of Naples, Naples, ITA
¹⁰U.O.C. Oncoematologia Pediatrica, Padova, ITA
¹¹Pediatric Hematology and Oncology Unit, A.R.N.A.S. Ospedale Civico, Palermo, Italy
¹²Ospedale Civile, Pescara, Italy
¹³Pediatric Oncology and Hematology "Lalla Seràgnoli", IRCCS Azienda Ospedaliero-Universitaria di Bologna, Bologna, Italy
¹⁴Department of Pediatrics, Institute of Molecular Medicine "Angelo Nocivelli," University of Brescia, Brescia, Italy
¹⁵Haematology Unit, Istituto Giannina Gaslini, Genoa, Italy., Genoa, Italy
¹⁶Department of Paediatrics, Paediatric Haematology-Oncology Unit, Fondazione MBBM/San Gerardo Hospital, Monza, Italy., Monza, ITA
¹⁷Department of Paediatrics, Paediatric Haematology-Oncology Unit, Fondazione MBBM/San Gerardo Hospital, Monza, Italy., Monza, AL, ITA
¹⁸Federico II University, Naples, NA, ITA
¹⁹Ospedale San Gerardo, Monza, MB, ITA
²⁰Centro Ricerca Tettamanti, Pediatric Department, University of Milano-Bicocca, Fondazione MBBM, Monza, MI, Italy
²¹Tettamanti Research Centre, Department of Paediatrics, University of Milano-Bicocca, Monza, Italy., Monza, ITA