Laura Raggi, PhD^1,2*, Annamaria Aprile, PhD^1,3, Simona Bolamperti, PhD^4*, Isabella Villa, PhD^4*, Mariangela Storto^1*, Sarah Marktel, MD^5* and Giuliana Ferrari, PhD^1,3*

¹San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy
²University of Milano Bicocca, Milan, Italy
³Vita-Salute San Raffaele University, Milan, Italy
⁴Endocrine & Osteometabolic Lab, IRCCS San Raffaele Scientific Institute, Milano, ITA
⁵Hematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, Milano, ITA

Ginette Balbin-Cuesta, MD, PhD^1,2, Greggory Myers, B.S.³, Zesen Lin, BS⁴, Claire Kerpet, B.S.^5*, Beth McGee^6*, Katherine Rybkin^7*, Richard King, M.D.⁸, Claire Drysdale, BSc^1,3, Ann Friedman, B.S.^5*, Lei Yu^3*, Masaki Ito, B.S.^9*, Rilie Saba^9*, Ayse Bilge Ozel, M.S., Ph.D.^10* and Rami Khoriaty, M.D.^3,5,11,12

¹Medical Scientist Training Program, University of Michigan Medical School, Ann Arbor, MI
²Cellular and Molecular Biology Program, University of Michigan Medical School, Northville, MI
³Department of Cell and Developmental Biology, University of Michigan Medical School, Ann Arbor, MI
⁴Department of Pharmacology, University of Michigan Medical School, Ann Arbor, MI
⁵Department of Internal Medicine, University of Michigan Medical School, Ann Arbor, MI
⁶Howard Hughes Medical Institute, Ann Arbor, MI
⁷College of Literature, Science, and the Arts, University of Michigan, Ann Arbor, MI
⁸Department of Internal Medicine, University of Michigan, Ann Arbor, MI
⁹College of Literature, Science, and Arts, University of Michigan, Ann Arbor, MI
¹⁰Department of Human Genetics, University of Michigan, Ann Arbor, MI
¹¹Cellular and Molecular Biology Program, University of Michigan Medical School, Ann Arbor, MI
¹²University of Michigan Rogel Cancer Center, Ann Arbor, MI

Elizabeth A. Traxler, MD, PhD^1,2, Chad Komar^3*, Megan Saari^2*, A Josephine Thrasher^4*, Kunhua Qin, PhD^2*, Eugene Khandros, MD, PhD², Quynn Hotan^4*, Osheiza Abdulmalik, DVM^2*, Claire Shao^5*, Qingzhou Chen, PhD^6*, Andy J. Minn, MD, PhD^7*, Cheryl A. Keller, PhD^8*, Junwei Shi, PhD^9* and Gerd A. Blobel, MD, PhD²

¹Division of Hematology/Oncology, Department of Medicine, Hospital of University of Pennsylvania, Philadelphia, PA
²Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA
³Perelman School of Medicine at University of Pennsylvania, Philadelphia
⁴Division of Hematology, Children's Hospital of Philadelphia, Philadelphia
⁵Department of Radiation Oncology, University of Pennsylvania, Philadelphia
⁶Department of Cancer Biology, Perelman School of Medicine at University of Pennsylvania, Philadelphia
⁷Department of Radiation Oncology, Perelman School of Medicine at University of Pennsylvania, Philadelphia, PA
⁸Department of Biochemistry and Molecular Biology, The Pennsylvania State University, University Park, PA
⁹Department of Cancer Biology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA

Alexis A. Thompson, MD, MPH^1,2, Timothy S. Olson, MD, PhD^1,3, Mark C. Walters, MD⁴, John B. Porter, MD^5*, Jennifer Schneiderman, MD, MS⁶, Suradej Hongeng, MD^7*, Andreas Kulozik, MD, PhD⁸, Marina Cavazzana, MD, PhD^9,10,11, Martin G. Sauer, MD¹², Adrian J. Thrasher, MD, PhD^13*, Isabelle Thuret, MD^14*, Ashutosh Lal, MD¹⁵, John E. J. Rasko, MBBS, PhD^16,17,18, Evangelia Yannaki, MD^19*, Shamshad Ali, MS, MStat^20*, Gloria Tao, PhD^20*, Himal L. Thakar, MD^20*, Ami Deora, PhD^20*, Katiana Gruppioni, MPH^20*, Richard A. Colvin, MD^20*, Franco Locatelli, MD, PhD^21,22 and Janet L. Kwiatkowski, MD, MSCE^1,23

¹Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA
²Division of Hematology, Children’s Hospital of Philadelphia, Philadelphia, PA
³Cellular Therapy and Transplant Section, Division of Oncology, Children's Hospital of Philadelphia, Philadelphia, PA
⁴UCSF Benioff Children's Hospital, Oakland, CA
⁵Research Department of Haematology, University College London Hospital, London, GBR
⁶Division of Hematology, Oncology, Neuro-oncology and Stem Cell Transplantation, Department of Pediatrics, Ann & Robert H. Lurie Children’s Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, IL
⁷Department of Pediatrics, Ramathibodi Hospital, Mahidol University, Bangkok, THA
⁸Department of Pediatric Oncology, Hematology, and Immunology and Hopp-Children’s Tumor Center, University of Heidelberg and DKFZ, Heidelberg, Germany
⁹Pediatric Immunology, Imagine Institute, Paris, FRA
¹⁰Department of Biotherapy, Hospital Necker, University Paris Descartes, Paris, France
¹¹Biotherapy Clinical Investigation Center, Inserm Assistance Publique Hôpitaux de Paris, Paris, France
¹²Department of Pediatric Hematology and Oncology, Hannover Medical School, Hannover, Germany
¹³Section of Molecular and Cellular Immunology, University College London Great Ormond Street Institute of Child Health, London, GBR
¹⁴Department of Pediatrics, Hospital Timone, Marseille, France
¹⁵Division of Hematology, UCSF Benioff Children’s Hospital, Oakland, CA
¹⁶Gene and Stem Cell Therapy Program, Centenary Institute, Camperdown, Australia
¹⁷Faculty of Medicine and Health, The University of Sydney, Sydney, Australia
¹⁸Department of Cell and Molecular Therapies, Royal Prince Alfred Hospital, Sydney, NSW, AUS
¹⁹Gene and Cell Therapy Center, Hematology Department-Hematopoietic Cell Transplantation Unit, G. Papanikolaou Hospital, Thessaloniki, Greece
²⁰bluebird bio, Inc., Somerville, MA
²¹Department of Pediatric Hematology and Oncology, IRCCS Ospedale Pediatrico Bambino Gesù, Rome, Italy
²²Department of Pediatrics, Catholic University of the Sacred Heart, Rome, Italy
²³Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA

Polyxeni Delaporta, MD, PhD^1,2*, Elena Chatzikalil, MD^1,2*, Vassilis Ladis, MD^1,2*, Maria Moraki, MD^1,2* and Antonis Kattamis, MD, PhD^1,2

¹'Aghia Sophia' Children's Hospital, ERN-EuroBloodNet Center, Athens, Athens, Greece
²Thalassemia Unit, First Department of Pediatrics, National and Kapodistrian University of Athens, Athens, Greece

Manuel Ugidos Guerrero, PhD^1*, Sujit Sheth, MD², Olivier Hermine^3,4 and Sadanand Vodala⁵

¹BMS Center For Innovation and Translational Research Europe (CITRE), Seville, Spain
²Division of Hematology and Oncology, Department of Pediatrics, Weill Cornell Medicine, New York, NY
³Imagine Institute, INSERM Unité 1163, University of Paris, Paris, France
⁴Department of Hematology, Necker Hospital, APHP, Paris, France
⁵Hematology, Translational Medicine and Disease Team, Bristol Myers Squibb, Summit, NJ

Lisa Bilston, MD¹, Kelsey Uminski, MD^1,2, Theodosia A. Kalfa, MD, PhD³, Ammar Husami^4*, M. Dawn Goodyear, MD, MSc^2,5 and Natalia Rydz, MD, FRCPC^1,2

¹Division of Hematology and Hematological Malignancies, Department of Medicine, University of Calgary, Calgary, Canada
²Southern Alberta Rare Blood and Bleeding Disorders Comprehensive Care Program, Calgary, Canada
³Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
⁴Division of Human Genetics, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH
⁵Department of Medicine, Division of Hematology and Hematological Malignancies, University of Calgary, Calgary, AB, Canada

Ruiqiong Wu, MD, PhD¹, Siqi Yi, Ph.D^2*, Yichao Li, PhD^3*, Dewan Shrestha^3*, Minzhang Zheng, PhD^3*, Jingjing Zhang^3* and Yong Cheng, PhD⁴

¹St. Jude Children Res. Hospital, Memphis, TN
²St. Jude Children's Research Hospital, Memphis, TN
³St. Jude Children Res. Hospital, Memphis, AL
⁴St. Jude Children's Rsesearch Hospital, Memphis, TN

Sarah Adams^1*, Anita Dhara^2*, Rachael White^2*, Archana Prabahar^1*, Peng Jiang^2*, Merlin Nithya Gnanapragasam, PhD² and Mahesh Ramamoorthy^2*

¹Cleveland State University, Cleveland
²Cleveland State University, Cleveland, OH