Aurelio Maggio, MD¹, Angela Vitrano, PhD^1*, Antonella Meloni, PhD^2*, Walter Addario Pollina^3*, Mehran Karimi, MD⁴, Amal El-Beshlawy, MD⁵, Mahmoud Hajipour^6*, Vito Di Marco, MD^7*, Saqib Hussain Ansari, MBBS, PhD^8*, Aldo Filosa, MD^9*, Paolo Ricchi, MD^9*, Adriana Ceci, MD^10*, Shahina Daar, MD^11*, Sylvia Titi Singer, MD¹², J F Borgio, MD^13*, Alessia Pepe, MD^2*, Salvatore Scondotto^3*, Gabriella Dardanoni^3*, Massimiliano Sacco, DR^1*, Laura Pistoia, PhD^2*, Rita Barone, MD^1*, Fedele Bonifazi^10*, Lorella Pitrolo, MD^1* and Elliott P. Vichinsky, MD¹²

¹Campus of Hematology Franco and Piera Cutino, AOOR Villa Sofia-V. Cervello, Palermo, Italy
²MRI Unit, Fondazione G. Monasterio CNR-Regione Toscana, Pisa, Italy
³Dipartimento Attività Sanitarie e Osservatorio Epidemiologico Assessorato Salute Regione Sicilia, Palermo, Italy
⁴Hematology Research Center, Nemazee Hospital, Shiraz University of Medical Science, Shiraz, Iran (Islamic Republic of)
⁵Pediatric Hospital, Cairo University, Mohandsein, Egypt
⁶Shiraz University of Medical Sciences, Shiraz, Iran (Islamic Republic of)
⁷Dipartimento Biomedico di Medicina Interna e Specialistica, University of Palermo, Palermo, Italy
⁸National Institute of Blood Disease and Bone Marrow Transplantation, KARACHI, OK, Pakistan
⁹Rare Blood Cell Disease Unit, AORN A. Cardarelli, Napoli, Italy
¹⁰Fondazione per la Ricerca Farmacologica Gianni Benzi Onlus, Valenzano (BA), Italy
¹¹Dept of Hematology, College of Medicine & Health Sciences, Sultan Qaboos University,, Muscat, Oman
¹²UCSF Benioff Children's Hospital Oakland, Oakland, CA
¹³Department of Genetic Research, Institute for Research and Medical Consultation (IRMC), Imam Abdulrahman Bin Faisal University, Dammam, Saudi Arabia

Eugene Khandros, MD, PhD¹, Peng Huang, PhD^1*, Scott A. Peslak, MD, PhD², Malini Sharma, BS^1*, Osheiza Abdulmalik, DVM³, Belinda Giardine^4*, Zhe Zhang, PhD^5*, Cheryl A. Keller, PhD^4*, Ross C. Hardison, Ph.D.^6* and Gerd A. Blobel, MD, PhD¹

¹Division of Hematology, Children's Hospital of Philadelphia, Philadelphia, PA
²Department of Medicine, Division of Hematology/Oncology, Hospital of the University of Pennsylvania, Philadelphia, PA
³Division of Hematology, Children’s Hospital of Philadelphia, Philadelphia, PA
⁴Department of Biochemistry and Molecular Biology, Center for Computational Biology & Bioinformatics, The Pennsylvania State University, University Park, PA
⁵Department of Biomedical and Health Informatics, Children's Hospital of Philadelphia, Philadelphia, PA
⁶Department of Biochemistry and Molecular Biology; Centers for Eukaryotic Gene Regulation and for Computational Biology, The Pennsylvania State University, University Park, PA

John B. Porter, MA, M.D., FRCP, FRCPath^1*, Alexis A. Thompson, MD^2,3, Mark C. Walters, MD⁴, Janet L. Kwiatkowski, MD, MSCE^5,6, Suradej Hongeng, MD⁷, Martin G. Sauer, MD⁸, Adrian J. Thrasher, MBBS, PhD, FMedSci^9*, Isabelle Thuret, MD^10*, Ruiting Guo, MS^11*, Richard A. Colvin, MD, PhD^11* and Franco Locatelli, MD, PhD¹²

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

Melih Acar^1*, Madhulika Jupelli^2*, Kyle MacBeth² and Martin Schwickart^2*

¹Formerly Bristol Myers Squibb, Princeton, NJ
²Bristol Myers Squibb, Princeton, NJ

Irene Motta, MD^1,2*, Valeria Maria Pinto, MD^3*, Filomena Longo, MD^4*, Federico Bonetti, MD^5*, Elisa De Michele, MD^6*, Alberto Piperno, MD^7*, Lorella Pitrolo, MD^8*, Micol Quaresima, MD^9*, Michela Ribersani, MD^10*, Margherita Migone De Amicis, MD^2*, Manuela Balocco, MD^3*, Barbara Gianesin, PhD^11*, Giovanna Graziadei, MD^2*, Maria Domenica Cappellini, MD¹, Lucia De Franceschi, MD¹², Antonio Piga, MD⁴ and Gian Luca Forni, MD^3*

¹Department of Clinical Sciences and Community Health, University of Milan, Milan, Italy
²Department of Internal Medicine, UOC Medicina Generale, Foundation IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy
³Hemoglobinopathies and Congenital Anemia Center, Ospedale Galliera, Genoa, Italy
⁴Department of Clinical and Biological Sciences, University of Turin, Turin, Italy
⁵Pediatric Haematology Oncology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy
⁶Medicina Trasfusionale, AUO "San Giovanni di Dio e Ruggi D'Aragona", Salerno, Italy
⁷Centre for Disorder of Iron Metabolism, ASST-Monza - S.Gerardo Hospital, Monza, Italy
⁸Campus of Hematology Franco and Piera Cutino, AOOR Villa Sofia-V. Cervello, Palermo, Italy
⁹Dipartimento di Medicina Diagnostica, Clinica e di Sanità Pubblica, University of Modena and Reggio Emilia, Modena, Italy
¹⁰UOC Haematology, Azienda Ospedaliera Universitaria Policlinico Umberto I di Roma, Rome, Italy
¹¹ForAnemia Foundation, Genoa, Italy
¹²Department of Medicine, Policlinico GB Rossi, University of Verona, Verona, Italy

Melih Acar^1*, Madhulika Jupelli^2*, Roberto A. Abbiati^3*, Harish N. Ramanathan^4*, Cristina C. Santini^1*, Alexander Ratushny^2*, Diana R. Dunshee^1*, Daniel Lopes de Menezes, PhD^2*, Kyle MacBeth², Rajasekhar N.V.S. Suragani⁴, Remco Loos^3* and Martin Schwickart^2*

¹Formerly Bristol Myers Squibb, Princeton, NJ
²Bristol Myers Squibb, Princeton, NJ
³BMS Center for Innovation and Translational Research Europe, Seville, Spain
⁴Acceleron Pharma, Cambridge, MA

Miao Lin^1*, Vijay Menon, PhD^2*, Raymond Liang, PhD^3*, Tasleem Arif, PhD^1*, Laura Breda, PhD^4,5*, Stefano Rivella, PhD⁵ and Saghi Ghaffari, MD, PhD¹

¹Department of Cell, Developmental and Regenerative Biology, Tisch Cancer Institute, Black Family Stem Cell Institute, Icahn School of Medicine at Mount Sinai, New York, NY
²Radiobiology, Yale School of Medicine, New Haven, CT
³Hemogenyx Pharmaceuticals LLC, Brooklyn, NY
⁴The Children's Hospital of Philadelphia, Philadelphia, PA
⁵Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA

Elena Spiteri, B.Sc^1*, Laura Grech, PhD, MSc, B.Sc^2*, Alexander E. Felice^2,3 and Joseph Borg, PhD, MSc, BSc⁴

¹Department of Applied Biomedical Science, Faculty of Health Sciences, University of Malta, Msida, Malta
²Thalassemia Testing and Hemoglobin Research Laboratory, and The Malta Bio-Bank, Department of Physiology and Biochemistry, University of Malta, Msida, Malta
³Division of Pathology, Thalassemia and Genetics Clinic, Mater Dei Hospital, Msida, Malta
⁴Department of Applied Biomedical Science, Faculty of Health Sciences, Mater Dei Hospital, Birkirkara, Malta