Maxwell Chappell^1*, Laura Breda, PhD^1*, Danuta Jadwiga Jarocha, PhD^1*, Michael Triebwasser, MD, PhD, MSc, BS^1,2, Tyler E Papp^3*, Valentina Ghiaccio, PhD^1*, Megan T Fedorky^1*, Amaliris Guerra, PhD^1*, Kandace Gollomp^1*, Osheiza Y. Abdulmalik, DVM^4*, Nattiya Teawtrakul, MD, PhD^5*, Stavros Glentis, PhD^6*, Antonis Kattamis, MD^6,7,8,9,10, Hamideh Parhiz, PhD, PharmD^3* and Stefano Rivella, PhD^{11,12,13,14,15}

¹Department of Pediatrics, Division of Hematology, The Children’s Hospital of Philadelphia, Philadelphia, PA
²U of Michigan, Ann Arbor, MI
³Division of Infectious Diseases, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA
⁴Division of Hematology, The Children’s Hospital of Philadelphia, Philadelphia, PA
⁵Srinagarind Khonkaen Hospital, Khonkaen, THA
⁶Pediatric Hematology Oncology Unit, First Department of Pediatrics, National and Kapodistrian University of Athens, Aghia Sophia Children’s Hospital, Athens, Greece
⁷Thalassemia Unit, First Department of Pediatrics, ‘Aghia Sophia’ Children’s Hospital, National and Kapodistrian University of Athens, ATHENS, Greece
⁸Division of Pediatric Hematology-Oncology, First Department of Pediatrics, National and Kapodistrian University of Athens, “Aghia Sophia” Children’s Hospital, Athens, Greece, ATHENS, Greece
⁹"Agia Sophia" Children Hospital, First Department of Pediatrics, National and Kapodistrian University of Athens, Athens, Greece, Athens, Greece
¹⁰National and Kapodistrian University of Athens, Athens, Greece
¹¹Department of Pediatrics, Division of Hematology, Children’s Hospital of Philadelphia, Philadelphia, PA
¹²Cell and Molecular Biology affinity group (CAMB), University of Pennsylvania, Philadelphia, PA
¹³Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA
¹⁴Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children’s Hospital of Philadelphia, Philadelphia, PA
¹⁵Penn Center for Musculoskeletal Disorders, University of Pennsylvania, Philadelphia, PA

Gabriele Casirati, MD^1,2,3*, Andrea Cosentino, MD^1,4,5*, Adele Mucci, PhD^1,6*, Iraxte Ugarte Zabala, BSc^1,7*, Mohammed S. Mahmoud, PhD^1,8*, Christian Brendel, PhD^9,10*, Alessandro Rambaldi, MD^11,12, Jerome Ritz, MD, PhD^13,14, Scott A. Armstrong, MD, PhD¹⁵ and Pietro Genovese, PhD^1,16,17

¹Dana Farber Cancer Institute, Boston, MA
²Universita' Milano Bicocca, milan, Italy
³Boston Children's Hospital, Boston
⁴Ospedale Papa Giovanni XXIII, Universita' di Studi di Milano, Bergamo, ITA
⁵Boston Children Hospital, Boston, MA
⁶Pediatric Oncology, Boston Children Hospital, Boston, MA
⁷German Cancer Research Center (DFKZ), Heidelberg, Germany
⁸Zoology department, Faculty of Science, Fayoum University, Fayoum, Egypt
⁹Hematology/Oncology, Harvard Medical School, Boston, MA
¹⁰Gene Therapy Program, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA
¹¹Hematology, ASST Papa Giovanni XXIII, Bergamo, Italy
¹²Department of Oncology and Hematology, University of Milan, Milan, Italy
¹³Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA
¹⁴Department of Medicine, Harvard Medical School, Boston, MA
¹⁵Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA
¹⁶Boston Children's Hospital, Boston, MA
¹⁷Harvard Medical School, Boston, MA

Laura Breda, PhD^1*, Tyler E Papp^2*, Michael Triebwasser, MD, PhD, MSc, BS^1,3, Amir Yadegari^2*, Megan T Fedorky^1*, Naoto Tanaka, PhD^1*, Houping Ni^2*, Drew Weissman, MD, PhD^2*, Stefano Rivella, PhD^4,5,6,7,8 and Hamideh Parhiz, PhD, PharmD^2*

¹Department of Pediatrics, Division of Hematology, The Children’s Hospital of Philadelphia, Philadelphia, PA
²Division of Infectious Diseases, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA
³U of Michigan, Ann Arbor, MI
⁴Department of Pediatrics, Division of Hematology, Children’s Hospital of Philadelphia, Philadelphia, PA
⁵Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA
⁶Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children’s Hospital of Philadelphia, Philadelphia, PA
⁷Cell and Molecular Biology affinity group (CAMB), University of Pennsylvania, Philadelphia, PA
⁸Penn Center for Musculoskeletal Disorders, University of Pennsylvania, Philadelphia, PA

Daisuke Araki, MD¹, Vicky Chen^2*, Neelam Redekar^3*, Poching Li^4*, Yan Luo^4*, Yuesheng Li^4* and Andre Larochelle, MD, PhD¹

¹Laboratory of Regenerative Therapies for Inherited Blood Disorders, Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institute, National Institutes of Health, Bethesda, MD
²Integrated Data Science Services, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD
³NIAID Collaborative Bioinformatic Resource (NCBR), NIH, Bethesda, MD
⁴DNA Sequencing and Genomics Core, National Heart Lung and Blood Institute, National Institutes of Health, Bethesda, MD

Byung-Chul Lee^1*, Max Grice^2*, Chuanfeng Wu, MD, PhD^3*, Ryland D Mortlock^4*, Taehoon Shin^5*, Yifan Zhou^5*, Uimook Choi, PhD^6*, Suk See De Ravin, MD, PhD^6*, So Gun Hong, DVM^5* and Cynthia E. Dunbar, MD⁵

¹NHLBI, NIH, BETHESDA, MD
²NIH, Bethesda, MD
³Translational Stem Cell Biology Branch, National Heart, Lung, and Blood Institute, NHLBI/NIH, Bethesda, MD
⁴Translational Stem Cell Biology Branch, National Heart Lung and Blood Institute, National Institutes of Health, Bethesda, MD
⁵Translational Stem Cell Biology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
⁶Laboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD

Jyoti Rana, PhD^1*, Sandeep RP Kumar, PhD^1*, Maite Munoz^1*, Kentaro Yamada, PhD^1*, Thais Bertollini, PhD^1*, David M Markusic, PhD¹, Manuela Corti, PhD^2*, Barry J Byrne, MD^2*, Roland W Herzog, PhD¹ and Moanaro Biswas, PhD^1*

¹Department of Pediatrics, IUPUI, Indianapolis, IN
²Powell Center for Rare Disease Research and Therapy, University of Florida, Gainesville, FL