Claudia R. Morris, MD^1,2, Nitya Bakshi, MBBS, MS³, Deborah Leake, CPNP^4*, Scott Gillespie, MS^5*, Lou Ann Scism Brown, PhD^6*, Frank Harris^6*, Kirshma Khemani, MD^7*, Alexus Locke, MS^8*, Christopher A Rees, MD, MPH^9*, Mark A Griffiths, MD^10*, Loretta Reyes, MD^11*, Polly Kumari, MBBS^12*, Sruti Shiva, PhD¹³ and Carlton D. Dampier, MD^2,14,15,16

¹Childrens Healthcare of Atlanta, Atlanta, GA
²Department of Pediatrics, Emory University School of Medicine, Atlanta, GA
³Department of Pediatrics/Aflac Cancer and Blood Disorders Center, Emory University, Atlanta, GA
⁴Pediatric Emergency Medicine, Children's Healthcare of/Emory University School of Medicine, Atlanta, GA
⁵Pediatric Biostatics Core, Emory University, Atlanta, GA
⁶Emory University School of Medicine, Atlanta, GA
⁷Department of Pediatrics, Division of Hematology/Oncology/BMT, Emory University School of Medicine, Atlanta, GA
⁸Emergency Medicine, Children’s Healthcare of Atlanta/Egleston Hospital, Atlanta, GA
⁹Pediatrics, Emory University School of Medicine, Atlanta, GA
¹⁰Emory University School of Medicine/Pediatric Emergency Medicine, Children’s Healthcare of Atlanta, Hughes Spalding, Atlanta, GA
¹¹Division of Pediatric Nephrology, Emory University School of Medicine/Children's Healthcare of Atlanta, Atlanta, GA
¹²Pediatrics Emergency Medicine, Emory University School of Medicine, Atlanta, GA
¹³Department of Pharmacology and Chemical Biology, University of Pittsburgh, Pittsburgh, PA
¹⁴Emory University, Atlanta, GA
¹⁵Aflac Cancer and Blood Disorders Center, Atlanta, GA
¹⁶Aflac Cancer and Blood Disorders Center, Children’s Healthcare of Atlanta, Atlanta, GA

Philippe Joly^1,2*, Elie Nader^3*, Flora Ketels^4*, Camille Boisson^5*, Romain Carin^6*, Celine Renoux, PhD^5,7*, Alexandra Gauthier^8*, Solène Poutrel^9*, Yves Bertrand, PhD^10* and Philippe Connes, PhD^11*

¹Groupement Hospitalier Est, Hospices Civils de Lyon, BRON, AL, France
²Laboratoire Interuniversitaire de Biologie de la Motricité (LIBM) - EA 7424, Université Claude-Bernard Lyon 1, Lyon, France
³Laboratoire Interuniversitaire de Biologie de la Motricité (LIBM) - EA7424, Université Claude-Bernard Lyon 1, LYON, France
⁴Laboratoire Interuniversitaire de Biologie de la Motricité (LIBM) - EA 7424, Université Calude-Bernard Lyon 1, LYON, France
⁵Laboratory LIBM EA7424, University of Lyon 1, “Vascular Biology and Red Blood Cell” team, Lyon, France
⁶Laboratoire Interuniversitaire de Biologie de la Motricité (LIBM) - EA 7424, Université Claude-Bernard Lyon 1, LYON, France
⁷Laboratory of Biochemistry and Molecular Biology, UF Biochemistry of Red Blood Cell diseases, Est Center of Biology and Pathology, Hospices Civils de Lyon, Lyon, France
⁸Institut d'Hématologie et d'Oncologie Pédiatrique (IHOP), LYON, France
⁹Hôpital Edouard-Herriot - Service de Médecine Interne, Hospices Civils de Lyon, LYON, France
¹⁰Institut d`Hematologie et d`Oncologie Pediatrique,Unité de coordination interne et externe, Lyon, France
¹¹Laboratoire LIBM EA7424 – Equipe « Biologie Vasculaire et du Globule Rouge », Université Claude Bernard Lyon 1, LYON, France

Myrthe J. van Dijk^1,2*, Sigrid van der Veen^2*, Minke A.E. Rab, MD, PhD^1,3*, Brigitte A. van Oirschot^1*, Jennifer Bos^1*, Cleo Derichs^2*, Anita W. Rijneveld, MD, PhD^4*, Marjon H. Cnossen^5*, Erfan Nur, MD, PhD^6,7, Bart J Biemond⁸, Marije Bartels^2*, Judith J.M. Jans^9*, Richard van Wijk^1* and Eduard J. Van Beers, MD, PhD^2*

¹Central Diagnostic Laboratory - Research, University Medical Center Utrecht, Utrecht University, Utrecht, Netherlands
²Center for Benign Hematology, Thrombosis and Hemostasis - Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, Netherlands
³Department of Hematology, University Medical Center Utrecht, Utrecht University, Utrecht, Netherlands
⁴Erasmus University Medical Center Cancer Institute, Rotterdam, Netherlands
⁵Department of Pediatric Hematology, Erasmus MC Sophia Children's Hospital, University Medical Center Rotterdam, Rotterdam, Netherlands
⁶Department of Blood Cell Research, Sanquin Research, Amsterdam, Netherlands
⁷Department of Hematology, Amsterdam University Medical Center, Amsterdam, Netherlands
⁸Department of Hematology, Amsterdam UMC location University of Amsterdam, Amsterdam, Netherlands
⁹Section Metabolic Diagnostics, Department of Genetics, University Medical Center Utrecht, Utrecht University, Utrecht, Netherlands

Modupe Idowu, MD¹, Thinh Nguyen, MD^2*, Derek Lewis, MD^3*, Thokozeni Lipato, MD^4*, Erin M. Buckley, PhD^5*, Rowan Brothers, BS^6*, Mariam Akbar, BS^7*, Sydney Triplett, BS^7*, Kobina Dufu, PhD^8* and Eleanor Lisbon, MD, MPH^8*

¹University of Texas McGovern Medical School, Houston, TX
²Visionaries Clinical Research, Atlanta, GA
³Arkansas Primary Care Clinic, Little Rock, AR
⁴Virginia Commonwealth University Health, Richmond, VA
⁵Department of Pediatrics, Emory University School of Medicine, Atlanta, GA
⁶Emory University, Atlanta, GA
⁷Wallace H. Coulter Department of Biomedical Engineering, Georgia Institute of Technology and Emory University, Atlanta, GA
⁸Global Blood Therapeutics, South San Francisco, CA

Mark C. Walters, MD¹, Alexis A. Thompson, MD, MPH^2,3, Janet L. Kwiatkowski, MD, MSCE^4,5, Suhag Parikh, MD^6*, Markus Y. Mapara, MD, PhD⁷, Stacey Rifkin-Zenenberg, DO⁸, Banu Aygun, MD⁹, Kimberly A. Kasow, DO^10*, Alex Miller, BS^11*, Lixin Zhang, PhD^11*, Anjulika Chawla, MD^11*, Elizabeth R. Macari, PhD^11*, Francis J. Pierciey Jr., MSc^11*, John F. Tisdale, MD^12* and Julie Kanter, MD¹³

¹UCSF Benioff Children's Hospital, Oakland, CA
²Northwestern University Feinberg School of Medicine, Chicago, IL
³Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL
⁴Department of Pediatrics, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA
⁵Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA
⁶Aflac Cancer and Blood Disorders Center, Department of Pediatrics, Children's Healthcare of Atlanta, Emory University, Atlanta, GA
⁷Division of Hematology/Oncology, Columbia Center for Translational Immunology, Columbia University Medical Center, New York, NY
⁸Hackensack University Medical Center, Hackensack, NJ
⁹Cohen Children’s Medical Center, New Hyde Park, NY
¹⁰University of North Carolina, Chapel Hill, NC
¹¹bluebird bio, Inc., Somerville, MA
¹²Cellular and Molecular Therapeutics Branch NHLBI/NIDDK, National Institutes of Health, Bethesda, MD
¹³University of Alabama Birmingham, Birmingham, AL

Haydar Frangoul, MD¹, Franco Locatelli, MD², Monica Bhatia, MD^3*, Markus Y. Mapara, MD, PhD⁴, Lyndsay Molinari, MD^5*, Akshay Sharma, MBBS⁶, Stephan Lobitz, MD^7*, Mariane de Montalembert, MD, PhD⁸, Damiano Rondelli, MD⁹, Martin Steinberg, MD¹⁰, Mark C. Walters, MD¹¹, Suzan Imren, MD^12*, Lanju Zhang, PhD^12*, Anjali Sharma, MD^13*, Yang Song, PhD^12*, Christopher Simard, MD^12*, William Hobbs, MD, PhD^12* and Stephen Grupp, MD^14*

¹Sarah Cannon Center Research Institute and The Children’s Hospital at TriStar Centennial, Nashville, TN
²Department of Pediatric Hematology and Oncology, Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome, Italy
³Department of Pediatrics, Columbia University, NewYork-Presbyterian Morgan Stanley Children’s Hospital, New York, NY
⁴Herbert Irving Comprehensive Cancer Center, Columbia University, New York, NY
⁵Sarah Cannon Pediatric Transplant and Cellular Therapy Program at Methodist Children’s Hospital, San Antonio, TX
⁶Bone Marrow Transplantation and Cellular Therapy, St. Jude Children’s Research Hospital, Memphis, TN
⁷Gemeinschaftsklinikum Mittelrhein, Koblenz, Germany
⁸Necker-Enfants Malades Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP), University of Paris, Paris, France
⁹University of Illinois at Chicago, Chicago, IL
¹⁰Boston University School of Medicine, Boston, MA
¹¹University of California San Francisco Benioff Children’s Hospital, Oakland, CA
¹²Vertex Pharmaceuticals Incorporated, Boston, MA
¹³CRISPR Therapeutics, Cambridge, MA
¹⁴Children’s Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA