M Domenica Cappellini¹, Vip Viprakasit², Yesim Aydinok³, John B Porter^4*, Yong-Rong Lai^5*, Noppadol Siritanaratkul², Zeynep Karakas^6*, Antonis Kattamis⁷, Candace Wang^8*, Zewen Zhu^8*, Felicity Schaefer^8* and Ali T Taher⁹

¹Universitá di Milano, Ca Granda Foundation IRCCS, Milan, Italy
²Department of Pediatrics and Internal Medicine, Siriraj Hospital, Mahidol University, Bangkok, Thailand
³Ege University Hospital, Izmir, Turkey
⁴University College London, London, United Kingdom
⁵The First Affiliated Hospital of Guangxi Medical University, Nanning, China
⁶Istanbul University, Istanbul Medical Faculty, Istanbul, Turkey
⁷First Dept of Pediatrics, University of Athens, Athens, Greece
⁸Novartis Pharmaceuticals, East Hanover, NJ
⁹American University of Beirut Medical Center, Beirut, Lebanon

Alessia Pepe^1*, Antonella Meloni^1*, Maria Giovanna Neri^1*, Massimo Allò^2*, Elena Facchini^3*, Tommaso Casini^4*, Aurelio Maggio, MD⁵, Vincenzo Positano^6*, Gianluca Valeri^7*, Elisabetta Chiodi^8*, Cristina Salvatori^9* and Maria Rita Gamberini^10*

¹CMR Unit, Fondazione G. Monasterio CNR-Regione Toscana, Pisa, Italy
²Servizio Microcitemia, Presidio Ospedaliero ASL 5, Crotone, Italy
³U.O. di Pediatria -Prof A. Pession, Azienda Ospedaliero-Universitaria di bologna - Policlinico "S. Orsola-MalpighiBologna - Policlinico S. Orsola-Malpighi", Bologna, Italy
⁴Centro Talassemie ed Emoglobinopatie, Ospedale Meyer, Firenze, Italy
⁵Unità Operativa Complessa Ematologia II, A.O.R. “Villa Sofia – V. Cervello’’, Palermo, Italy
⁶Unità Operativa Complessa Ematologia con Talassemia, “ARNAS” Civico, Palermo, Italy
⁷Dipartimento di Radiologia, Azienda Ospedaliero-Universitaria Ospedali Riuniti "Umberto I-Lancisi-Salesi", Ancona, Italy
⁸Servizio Radiologia Ospedaliera-Universitaria, Arcispedale “S.Anna”, Ferrara, Italy
⁹Unità Operativa Sistemi Informatici, Fondazione G. Monasterio CNR-Regione Toscana, Pisa, Italy
¹⁰Pediatria, Adolescentologia e Talassemia, Arcispedale “S.Anna”, Ferrara, Italy

Debmalya Bhattacharyya^1*, Maitreyee Bhattacharyya, DM¹, Saswata Chatterjee^2*, Abhijit Chowdhury^2* and Pramit Ghosh^3*

¹Institute of Haematology and Transfusion Medicine,Medical College, Kolkata, India
²School of Digestive and Liver Diseases, IPGMER, Kolkata, India
³Department of Community Medicine, Medical College, Kolkata, India

Andrea Jarisch, MD^1*, Regine Grosse, MD^2*, Emilia Salzmann-Manrique^3*, Roland Fischer, PhD^4*, Jan Soerensen, MD^3*, Renate Hammerstingl, MD^5*, Eva Rettinger, MD^3*, Andre Manfred Willasch, MD^6*, Thomas Klingebiel, MD⁷ and Peter Bader, MD⁸

¹Division for Stem Cell Transplantation and Immunology, Hospital for Children and Adolescents, University Hospital Frankfurt, Frankfurt am Main, Germany
²University Medical Center Hamburg-Eppendorf/Pediatric and Adult Hematology and Oncology, University, Hamburg, Germany
³Division for Stem Cell Transplantation and Immunology, Hospital for Children and Adolescents, University Hospital Frankfurt, Frankfurt, Germany
⁴University Medical Center Hamburg-Eppendorf/Pediatric Hematology and Oncology, University, Hamburg, Germany
⁵Institute for Diagnostic and Interventional Radiology, University Hospital Frankfurt, Frankfurt, Germany
⁶Division for Stem Cell Transplantation and Immunology, Hospital for Children and Adolescents, University Hospital Frankfurt, Frankfurt Am Main, Germany
⁷Division for Stem Cell Transplantation and Immunology, Department for Children and Adolescents, University Hospital Frankfurt, Goethe University, University Hospital Frankfurt, Frankfurt am Main, Germany
⁸Division for Stem Cell Transplantation and Immunology, Hospital for Children and Adolescents, University of Frankfurt, Frankfurt, Germany

Pierre-Olivier Gaudreau, M.D., M.Ps., FRCPC^1*, Xiaoduan Weng, M.D., Ph.D.^1*, Marilyn Brillant, T.M.^1*, Michael Newmarch, M.D.², Guillaume Lettre, Ph.D.^3*, Michele Mahone, M.D., FRCPC^4*, Florence Weber, M.D., FRCPC^4*, Marie-Josee Bedard, M.D., FRCSC^5*, Andre Masse, M.D., FRCSC^5*, John Stagg, Ph.D.^6*, Vincent-Philippe Lavallee, M.D.⁷ and Denis Soulieres, MD, FRCPC, MSc¹

¹Hematology and Medical Oncology, Notre-Dame Hospital (CHUM), Montreal, QC, Canada
²Hematology, Maisonneuve-Rosemont Hospital, Montreal, QC, Canada
³Montreal Heart Institute, Universite de Montreal, Montreal, QC, Canada
⁴Internal Medicine, Saint-Luc Hospital (CHUM), Montreal, QC, Canada
⁵Obstetrics and Gynecology, Saint-Luc Hospital (CHUM), Montreal, QC, Canada
⁶CHUM Research Center (CRCHUM), Montreal, QC, Canada
⁷Institute for Research in Immunology and Cancer, Université de Montréal, Montreal, QC, Canada

N. Scott Reading, Ph.D.^1,2,3*, Barnaby E. Clark, Ph.D.^4*, Jihyun Song, MS^2*, Claire C. Shooter^5*, Robin E. Miller, M.D.⁶, Archana M Agarwal, M.D.^3*, Swee Lay Thein, M.D.^7,8, Vladimir Divoky, PhD⁹ and Josef T. Prchal, M.D.^1,10,11

¹Institute for Clinical and Experimental Pathology, ARUP Laboratories, Salt Lake City, UT
²Division of Hematology, School of Medicine, University of Utah, Salt Lake City, UT
³Department of Pathology, University of Utah, Salt Lake City, UT
⁴Department of Molecular Pathology, Viapath at King's College Hospital, London, United Kingdom
⁵Molecular Haematology, Division of Cancer Studies, King's College London, London, United Kingdom
⁶Nemours Center for Cancer and Blood Disorders, Nemours/Al duPont Hospital for Children, Wilmington, DE
⁷Molecular Haematology, Division of Cancer Studies, Kings College London, London, United Kingdom
⁸National Heart, Lung and Blood Institute, Sickle Cell Branch, National Institutes of Health, Bethesda, MD
⁹Department of Biology, Faculty of Medicine and Dentistry, Palacky University, Olomouc, Czech Republic
¹⁰Division of Hematology, University of Utah, Salt Lake City, UT
¹¹Department of Medicine, George E. Wahlen Department of Veterans Affairs Medical Center, Salt Lake City, UT

Daniel Garcia dos Santos, PhD^1*, Zuzana Zidova, MSc^2*, Marc Mikhael, PhD^3*, Amel Hamdi, PhD^4*, Monika Horvathova, PhD⁵ and Prem Ponka, MD, PhD, FCMA¹

¹Lady Davis Institute for Medical Research, Montreal, QC, Canada
²Faculty of Medicine and Dentistry, Palacky University, Olomouc, Czech Republic
³School of Arts and Science, Lebanese American University, Beirut, Lebanon
⁴Department of Physiology and Medicine, McGill University, Montreal, QC, Canada
⁵Department of Biology, Faculty of Medicine and Dentistry, Palacky University, Olomouc, Czech Republic

Molly Susan Hein, BS^1*, Jennifer L Oliveira, MD¹, Kenneth C Swanson, MT(ASCP)^1*, Patrick A Lundquist, MB(ASCP), CG(ASCP)^2*, Joella A Yungerberg, MLS(ASCP)^1*, Lea M Coon, MS^1*, Brian D Dawson, PhD^2*, Ronald S. Go, MD³, Dragan Jevremovic, MD PhD^1* and James D Hoyer, MD¹

¹Hematopathology and Metabolic Hematology Laboratory, Mayo Clinic, Rochester, MN
²Laboratory Medicine and Pathology, Mayo Clinic, Rochester, MN
³Division of Hematology, Mayo Clinic, Rochester, MN

Barnaby E. Clark, Ph.D.^1,2*, Claire C. Shooter^2*, Frances Smith^1*, David Brawand^1*, Laura Steedman^1*, Matthew Oakley^1*, Peter Rushton^1*, Helen Rooks^3*, Xunde Wang, PhD^4*, Anthi Drousiotou^5*, Andriani Kyrri^6*, Xenia Felekis^5*, Miranda Petrou^5*, Andrew M Will, MD^7*, Chris Fisher, Ph.D.^8*, Douglas R. Higgs, FRS⁸, Cornelius Harteveld^9*, Marion Phylipsen^9*, Marina Kleanthous, PhD^5* and Swee Lay Thein, MD, DSc^2,10

¹Department of Molecular Pathology, Viapath at King's College Hospital, London, United Kingdom
²Molecular Haematology, Division of Cancer Studies, King's College London, London, United Kingdom
³Molecular Haematology, King’s College London, London, United Kingdom
⁴Sickle Cell Vascular Disease Section, Hematology Branch, National Heart, Lung, and Blood Institute, Bethesda, MD
⁵The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus
⁶Thalassaemia Center, Nicosia, Cyprus
⁷Paediatric Haematology, Royal Manchester Children’s Hospital, Manchester, United Kingdom
⁸MRC Molecular Haematology Unit, Weatherall Institute of Molecular Medicine, Oxford University, Oxford, United Kingdom
⁹Department of Clinical Genetics, Leiden University Medical Center, Leiden, Netherlands
¹⁰National Heart, Lung and Blood Institute, Sickle Cell Branch, National Institutes of Health, Bethesda, MD

Ciaran Lee, PhD^1*, Gang Bao, PhD^1*, Matthew H. Porteus, MD, PhD², Tatjana Cornu, PhD^3*, Annarita Miccio, PhD^4,5*, Thomas Cradick^6*, Toni Cathomen, PhD^3*, Ante Lundberg, MD⁷ and Fulvio Mavilio, PhD^4*

¹Rice University, Houston, TX
²Department of Pediatrics, Cancer Biology, Stanford University School of Medicine, Stanford, CA
³University Medical Center Freiburg, Freiburg, Germany
⁴Genethon, Evry, France
⁵Imagine Institute, Paris, France
⁶CRISPR Therapeutics, Boston, MA
⁷CRISPR THERAPEUTICS, CAMBRDIGE, MA

Carolina A Braghini, MSc^1,2*, Fernando F Costa, PhD, MD², Flavia C Costa, PhD^3*, Halyna Fedosyuk, MS^1*, Matthew Parker, BS^1*, Allen M Chazelle, MS^1* and Kenneth R Peterson, PhD^1,4

¹Biochemistry and Molecular Biology, University of Kansas Medical Center, Kansas City, KS
²Hematology and Hemotherapy Center, University of Campinas - SP, Sao Paulo, Brazil
³Kansas Biosciences Authority, Olathe, KS
⁴Anatomy and Cell Biology, University of Kansas Medical Center, Kansas City, KS

Jeffrey R Shearstone, PhD¹, Apurva Chonkar, MS^1*, Kailash Bhol, PhD^1*, Simon S Jones, PhD^2* and Matt Jarpe, PhD^1*

¹Acetylon Pharmaceuticals, Inc., Boston, MA
²Acetylon Pharmaceuticals, Inc., Boston