Janet L. Kwiatkowski, MD, MSCE^1,2, Mark C. Walters, MD³, Suradej Hongeng, MD⁴, Franco Locatelli, MD, PhD⁵, John E.J. Rasko, BSc, MBBS, PhD^6,7,8*, Marina Cavazzana, MD, PhD^9,10,11, Ying Chen, PhD^12*, Richard A. Colvin, MD, PhD^12* and Alexis A. Thompson, MD^13,14

¹Division of Hematology, Children's Hospital of Philadephia, Philadelphia, PA
²Department of Pediatrics, Perelman School of Medicine of the University of Pennsylvania, Philadelphia, PA
³UCSF Benioff Children's Hospital Oakland, Oakland, CA
⁴Mahidol University, Ramathibodi Hospital, Bangkok, Thailand
⁵Department of Pediatric Hematology/Oncology, IRCCS Ospedale Pediatrico Bambino Gesù, Rome, Italy
⁶Royal Prince Alfred Hospital, Camperdown, Australia
⁷Sydney Medical School, University of Sydney, Sydney, Australia
⁸Gene and Stem Cell Therapy Program, Centenary Institute, Camperdown, Australia
⁹Necker Children’s Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France
¹⁰IMAGINE Institute, Université Paris Descartes, Sorbonne Paris Cité, Paris, France
¹¹Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, Paris, France
¹²bluebird bio, Inc., Cambridge, MA
¹³Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL
¹⁴Northwestern University Feinberg School of Medicine, Chicago, IL

Alexis A. Thompson, MD^1,2, Janet L. Kwiatkowski, MD, MSCE^3,4, John B. Porter, MA, M.D., FRCP, FRCPath^5*, Suradej Hongeng, MD⁶, Evangelia Yannaki, MD^7*, Andreas E. Kulozik, MD, PhD^8,9*, Martin G. Sauer, MD¹⁰, Adrian J. Thrasher, MBBS, PhD, FMedSci^11*, Isabelle Thuret, MD^12*, Ashutosh Lal, MD¹³, Ruiting Guo, MS^14*, Weijian Liu, PhD^14*, Richard A. Colvin, MD, PhD^14*, Mark C. Walters, MD¹³ and Franco Locatelli, MD, PhD¹⁵

¹Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL
²Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, IL
³Division of Hematology, Children's Hospital of Philadephia, Philadelphia, PA
⁴Department of Pediatrics, Perelman School of Medicine of the University of Pennsylvania, Philadelphia, PA
⁵Haematology Department, University College London Hospitals, London, United Kingdom, United Kingdom
⁶Mahidol University, Ramathibodi Hospital, Bangkok, Thailand
⁷Hematology Department and HCT Unit, George Papanikolaou General Hospital, Thessaloniki, Greece
⁸Department of Pediatric Oncology, Hematology, and Immunology, University of Heidelberg, Heidelberg, Germany
⁹Molecular Medicine Partnership Unit (MMPU), European Molecular Biology Laboratory (EMBL), University of Heidelberg, Heidelberg, Germany
¹⁰Pediatric Hematology and Oncology, Medizinische Hochschule Hannover, Hannover, Germany
¹¹UCL Great Ormond Street Institute of Child Health, London, United Kingdom
¹²Pediatric Hematology, Hôpital de la Timone, Marseille, France
¹³UCSF Benioff Children's Hospital Oakland, Oakland, CA
¹⁴bluebird bio, Inc., Cambridge, MA
¹⁵Department of Pediatric Hematology/Oncology, Cell and Gene Therapy, IRCCS Bambino Gesù Children’s Hospital, Rome, Italy

Kunhua Qin^1*, Peng Huang, PhD^1*, Anran Huang, BS^1*, Ruopeng Feng, PhD², Thiyagaraj Mayuranathan^3*, Cheryl A. Keller, PhD^4*, Belinda Giardine^4*, Ross C. Hardison, Ph.D.^5*, Mitchell J. Weiss, MD, PhD³, Junwei Shi, PhD^6* and Gerd A. Blobel, MD, PhD¹

¹Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA
²Department of Hematology, St. Jude Children’s Research Hospital, Memphis, TN
³Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN
⁴Department of Biochemistry and Molecular Biology, Center for Computational Biology & Bioinformatics, The Pennsylvania State University, University Park, PA
⁵Department of Biochemistry and Molecular Biology; Centers for Eukaryotic Gene Regulation and for Computational Biology, The Pennsylvania State University, University Park, PA
⁶Department of Cancer Biology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA

Xianjiang Lan, PhD^1*, Ren Ren, PhD^2*, Ruopeng Feng, PhD³, Lana C Ly^4*, Yemin Lan, PhD^5*, Zhe Zhang, PhD^6*, Nicholas Aboreden^5*, Kunhua Qin^1*, John R Horton^2*, Jeremy D. Grevet, PhD^7*, Thiyagaraj Mayuranathan^8*, Osheiza Abdulmalik, DVM⁹, Cheryl A. Keller, PhD^10*, Belinda Giardine^11*, Ross C. Hardison, Ph.D.^12*, Merlin Crossley, PhD^4*, Mitchell J. Weiss, MD, PhD⁸, Xiaodong Cheng, PhD^2*, Junwei Shi, PhD^13* and Gerd A. Blobel, MD, PhD^1,5

¹Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA
²University of Texas MD Anderson Cancer Center, Houston
³Department of Hematology, St. Jude Children’s Research Hospital, Memphis, TN
⁴University of New South Wales, Sydney, Australia
⁵University of Pennsylvania, Philadelphia, PA
⁶Department of Biomedical and Health Informatics, Children's Hospital of Philadelphia, Philadelphia, PA
⁷Massachusetts General Hospital, Boston
⁸Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN
⁹Division of Hematology, Children's Hosp. of Phila., Philadelphia, PA
¹⁰Pennsylvania State University, University Park
¹¹Department of Biochemistry and Molecular Biology, Center for Computational Biology & Bioinformatics, The Pennsylvania State University, University Park, PA
¹²Department of Biochemistry and Molecular Biology; Centers for Eukaryotic Gene Regulation and for Computational Biology, The Pennsylvania State University, University Park, PA
¹³Department of Cancer Biology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA

Divya S Vinjamur, PhD^1*, Qiuming Yao, PhD^2*, Mitchel A. Cole, BS^3*, Connor McGuckin^4*, Chunyan Ren, PhD^2*, Jing Zeng, MD^2*, Mir Hossain, PhD^1*, Luca Pinello, PhD^5* and Daniel E Bauer, MD, PhD⁶

¹Division of Hematology and Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA
²Division of Hematology and Oncology, Boston Children’s Hospital, Harvard Medical School, Boston, MA
³Division of Hematology and Oncology, Boston Children’s Hospital, Boston, MA
⁴Division of Hematology and Oncology, Boston Children's Hospital, Boston, MA
⁵Molecular Pathology Unit and Center for Cancer Research, Massachusetts General Hospital, Charlestown, MA
⁶Division of Hematology and Oncology, Boston Children's Hospital / Dana-Farber Cancer Institute, Harvard Medical School, Cambridge, MA

Annamaria Aprile, PhD¹, Laura Raggi^1*, Mariangela Storto^1*, Isabella Villa, PhD^2*, Sarah Marktel, MD^3*, Irene Motta, MD^4,5*, Maria Domenica Cappellini^6,7, Alessandro Rubinacci, MD^2* and Giuliana Ferrari, PhD^8,9

¹San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy
²Bone Metabolism Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy
³Haematology and BMT Unit, IRCCS San Raffaele Scientific Institute, Milan, Italy
⁴Department of Internal Medicine, UOC Medicina Generale, Foundation IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy
⁵University of Milan, Milan, Italy
⁶Department of Clinical Sciences and Community Health, University of Milan, Milan, Italy
⁷Foundation IRCCS Ca' Granda Policlinico Milano, Milan, Italy
⁸San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCCS San Raffaele Scientific Institute, Milan, Italy
⁹Vita-Salute San Raffaele University, Milan, Italy