Qiaozhi Wei^1* and Paul S. Frenette, MD²

¹Albert Einstein College of Medicine, Bronx, NY
²Ruth L. and David S. Gottesman Institute for Stem Cell and Regenerative Medicine Research, Albert Einstein College of Medicine, Bronx, NY

Don M. Wojchowski, PhD¹, Edward Jachimowicz^1*, Ruth Asch^2* and Nicole M. Rainville^2*

¹COBRE Center of Excellence in Stem & Progenitor Cell Biology and Regenerative Medicine, Maine Medical Center Research Institute, Scarborough, ME
²Maine Medical Center Research Institute, Scarborough, ME

Betsabeh Khoramian Tusi, Ph.D.^*, Daniel Hidalgo, MA^* and Merav Socolovsky, MBBS, PhD

Dept. of Molecular, Cell and Cancer Biology, University of Massachusetts Medical School, Worcester, MA

Nobuto Arashiki, PhD^1*, Yuichi Takakuwa, MD, PhD^1*, Hiromi Ogura, MD, PhD^2*, Taiju Utsugisawa, MD, PhD^2*, Satoru Miyano, MD, Ph.D.^3*, Seishi Ogawa, MD, Ph.D.⁴, Seiji Kojima, MD, PhD^5*, Shouichi Ohga, MD. PhD^6*, Narla Mohandas, DSc⁷ and Hitoshi Kanno, MD, PhD^2,8*

¹Dept. Biochemistry, Tokyo Women's Medical University, Tokyo, Japan
²Dept. Transfusion Medicine and Cell Processing, Tokyo Women's Medical University, Tokyo, Japan
³Human Genome Center, Institute of Medical Science, The University of Tokyo, Tokyo, Japan
⁴Department of Pathology and Tumor Biology, Graduate School of Medicine, Kyoto University, Kyoto, Japan
⁵Department of Pediatrics, Nagoya University Graduate School of Medicine, Nagoya, Japan
⁶Department of Pediatrics, Yamaguchi University, Ube, Japan
⁷New York Blood Center, New York, NY
⁸Division of Genomic Medicine, Department of Advanced Biomedical Engineering and Science, Graduate School of Medicine, Tokyo Women's Medical University, Tokyo, Japan

Immacolata Andolfo^1*, Roberta Russo^1*, Francesco Manna^1*, Marica Lisa Salve^1*, Alok K. Sharma^2*, Seth L. Alper^2*, Lucia De Franceschi, MD³ and Achille Iolascon, MD, PhD⁴

¹CEINGE, Biotecnologie Avanzate, Naples, Italy, Department of Molecular Medicine and Medical Biotechnologies, “Federico II” University of Naples, Naples, Italy, Napoli, Italy
²Renal Division and Vascular Biology Research Center, Beth Israel Deaconess Medical Center and Department of Medicine, Harvard Medical School, Boston, MA 02215, USA., Boston, MA
³Department of Medicine, University of Verona and AOUI-Verona, Verona, Italy
⁴Department of Molecular Medicine and Medical Biotechnologies, “Federico II” University of Naples, Naples, Italy

Antonino Giambona^1*, Gianfranca Damiani^2*, Filippo Leto^1*, Cristina Yakil^2*, Cristina Passarello^1*, Valentina Cigna^2*, Giovanna Schillaci^2*, Massimiliano Sacco^1*, Rosario Di Maggio^1*, Angela Vitrano^1* and Aurelio Maggio¹

¹Campus of Haematology Franco e Piera Cutino- A.O.O.R. “Villa Sofia-Cervello”, Palermo, Italy
²U.O.S. Prenatal Diagnosis, Campus of Haematology Franco e Piera Cutino- A.O.O.R. “Villa Sofia-Cervello”, Palermo, Italy

Hongyu Wu, PhD^1*, Mikhail Bogdanov, PhD^2*, Yujin Zhang, MD^3*, Kaiqi Sun, BS^3,4, Anren Song, PhD^2*, Hong Liu, MD^3*, Morayo G, Adebiyi^2*, Danny C. Alexander^5*, Michael V. Milburn^5*, Modupe Idowu, MD⁶, Harinder S. Juneja, MD⁶ and Yang Xia, MD, Ph.D^4,7

¹Univeristy of Texas-Medical School, Houston, TX
²University of Texas-Medical School, Houston, TX
³Department of Biochemistry and Molecular Biology, UTHSC-Houston Medical School, Houston, TX
⁴University of Texas Graduate School of Biomedical Science, Houston, TX
⁵3Metabolon, Inc.,, Durham, NC
⁶Department of Internal Medicine-Hematology Division, University of Texas Health Science Center at Houston, Houston, TX
⁷Department of Biochemistry and Molecular Biology, University of Texas Medical School at Houston, Houston, TX

Eliza B Geer, MD^1*, Lilian Varricchio, PhD^2*, Fabrizio Martelli, PhD^3*, Wu He, PhD^4*, Lizette Couto^5*, Yelena Lazar^6*, Vanessa Cohen^6* and Anna Rita Migliaccio, PhD²

¹Mount Sinai Pituitary Care and Research Center, Icahn School of Medicine at Mount Sinai, New York, NY
²Tish Cancer Institute, Mount Sinai School of Medicine, New York, NY
³National AIDS Center, Istituto Superiore Di Sanita', Rome, Italy
⁴Icahn School of Medicine at Mount Sinai, Flow Cytometry Shared Resource Facility, New York, NY
⁵Department of Medicine & Neurosurgery, Icahn School of Medicine at Mount Sinai, New York, NY
⁶Icahn School of Medicine at Mount Sinai, New York, NY

Rachael F. Grace, MD¹, D Holmes Morton, MD^2*, Wilma Barcellini, MD^3*, Stefan W. Eber, MD⁴, Jenny M Depsotovic, DO^5*, Christine M. Knoll, MD⁶, Hassan M. Yaish, MD^7*, Peter E. Newburger, MD⁸, Jennifer Rothman, MD⁹, Alexis A. Thompson, MD¹⁰, Yaddanapudi Ravindranath, MBBS¹¹, Joachim Kunz, MD^12*, Eduard J van Beers, MD, PhD^13*, Marcin W Wlodarski, MD^14*, Kerri A Nottage, MD, MPH¹⁵, Heng Wang, MD^16*, Mukta Sharma, MD, MPH¹⁷, Melissa J. Rose, DO¹⁸, Yves D. Pastore, MD¹⁹, Patricia J. Giardina, MD²⁰, Melissa Rhodes, MD²¹, Kevin Kuo, BSc, MD²², Vicky R. Breakey, BSc, MD, MEd, FRCPC²³, Ellis J. Neufeld, MD, PhD¹, Dongjing Guo, MS^1*, Wendy B. London, PhD^1*, Jill Falcone^1* and Bertil Glader, MD, PhD²⁴

¹Harvard Medical School, Dana Farber Boston Children’s Cancer and Blood Disorder Center, Boston, MA
²Lancaster General Hospital, Clinic for Special Children, Lancaster, PA
³Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Milan, Italy
⁴Schwerpunktpraxis für Pädiatrische Hämatologie- Onkologie and Technical University, Munich, Germany
⁵Baylor College of Medicine, Texas Children’s Hematology Center, Houston, TX
⁶Phoenix Children's Hospital, Phoenix, AZ
⁷Primary Children’s Hospital, University of Utah, Salt Lake City, UT
⁸University of Massachusetts Medical School, Worcester, MA
⁹Duke University Medical Center, Durham, NC
¹⁰Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL
¹¹Wayne State University School of Medicine, Children’s Hospital of Michigan, Detroit, MI
¹²Heidelberg University, Heidelberg, Germany
¹³Van Creveldkliniek, University Medical Center, Utrecht, Netherlands
¹⁴University Children´s Hospital Freiburg, Division of Pediatric Hematology and Oncology, University of Freiburg, Freiburg, Germany
¹⁵St. Jude Children's Research Hospital, Memphis, TN
¹⁶DDC Clinic for Special Needs Children, Middlefield, OH
¹⁷Children's Mercy Hospital, Kansas City, MO
¹⁸The Ohio State University College of Medicine, Nationwide Children's Hospital, Columbus, OH
¹⁹CHU Sainte-Justine, Montreal, QC, Canada
²⁰Weill Cornell Medical College, New York Presbyterian Hospital, New York, NY
²¹University of Mississippi Medical Center, Jackson, MS
²²University of Toronto, University Health Network, Ontario, Canada
²³Department of Pediatrics, McMaster University, Hamilton, ON, Canada
²⁴Stanford University, Lucile Packard Children’s Hospital, Palo Alto, CA

Antonio M Risitano, MD, PhD¹, Michael Storek^2*, Leonardo Sahelijo, MD^2*, Mittie Doyle^2*, Yang Dai^2*, Ilene Ceil Weitz, MD³, Judith C.W. Marsh, MD⁴, Modupe Elebute, MBChB, MD^5*, Casey L O'Connell, MD⁶, Austin G. Kulasekararaj, MBBS, MD, MRCP, FRCPath^7*, Giridharan Ramsingh, M.D⁸, Serena Marotta, MD^1*, Andrzej Hellmann⁹ and Ante S. Lundberg, MD²

¹Hematology, Department of Clinical Medicine and Surgery, Federico II University of Naples, Naples, Italy
²Alexion Pharmaceuticals, Inc., Cheshire, CT
³Jane Anne Nohl Division of Hematology, University of Southern California-Keck School of Medicine, Los Angeles, CA
⁴King's College Hospital NHS Foundation Trust, London, United Kingdom
⁵Department of Haematology, King's College Hospital, London, United Kingdom
⁶Norris Comprehensive Cancer Center, Jane Anne Nohl Division of Hematology, University of Southern California, Los Angeles, CA
⁷Department of Haematological Medicine, Kings College Hospital NHS Foundation Trust, London, United Kingdom
⁸Hematology, University of Southern California, Los Angeles, CA
⁹Department of Hematology, Medical University of Gdansk, Gdansk, Poland

Yasutaka Ueda, MD, PhD¹, Makiko Osato, DS^1*, Wynne Weston-Davies, MB FRCS^2*, Miles A Nunn, DPhil^2*, Satoru Hayashi^1*, Jun-Ichi Nishimura, MD, PhD¹ and Yuzuru Kanakura, MD, PhD¹

¹Department of Hematology and Oncology, Osaka University Graduate School of Medicine, Suita, Japan
²Volution Immuno Pharmaceuticals (UK) Ltd, London, United Kingdom

Marta Subías-Hidalgo, PhD^1*, Alicia Lopez, PhD^1*, Jaouad Anter, PhD^1*, Sheila Pinto, PhD^1*, Emilio Ojeda, MD^2*, Jose A Garcia-Marco, MD, PhD², Fernando Ataulfo Gonzalez-Fernandez, MD^3*, Rafael Fores, MD^2*, Laura Llorente, MD^2*, Ana Maria Villegas-Martinez, MD^3* and Santiago Rodriguez-Cordoba, MD^1*

¹Centro de Investigaciones Biológicas, CSIC. Madrid, Spain., Patología Molecular/Biologica del Complemento, Madrid, Spain
²Hematology, University Hospital Puerta de Hierro Majadahonda, Majadahonda, Spain
³Hematology, Hospital Clínico San Carlos. Madrid, Spain, Madrid, Spain

Jennifer L Oliveira, MD¹, Lori A Frederick, BS^2*, Lea M Coon, MS^1*, Molly Susan Hein, BS^1*, Mrinal M Patnaik, MBBS³, Ayalew Tefferi, MD⁴, Animesh Pardanani, MBBS, PhD⁵, Stefan K Grebe, M.D., Ph.D.^6*, David S Viswanatha, MD² and James D Hoyer, MD¹

¹Hematopathology and Metabolic Hematology Laboratory, Mayo Clinic, Rochester, MN
²Hematopathology, Mayo Clinic, Rochester, MN
³Division of Hematology, Mayo Clinic, Rochester, MN
⁴Division of Hematology, Department of Internal Medicine, Mayo Clinic, Rochester, MN
⁵Division of Hematology, Department of Medicine, Mayo Clinic, Rochester, MN
⁶Laboratory Medicine and Pathology, Mayo Clinic, Rochester, MN

Angela Vitrano^1*, Giuseppina Calvaruso^1*, Eliana Lai^2*, Grazia Colletta^3*, Alessandra Quota^4*, Calogera Gerardi^5*, Luciana Concetta Rigoli^6*, Liana Cuccia^7*, Francesco Gagliardotto^8*, Aldo Filosa, MD^9*, Vincenzo Caruso, MD^10*, Crocetta Argento^11*, Saveria Campisi^12*, Michele Rizzo, MD^13*, Carmelo Fidone^14*, Paolo Rigano^1*, Massimiliano Sacco^1*, Alessia Pepe^15*, Rosario Di Maggio^1*, Lorella Pitrolo^1* and Aurelio Maggio¹

¹Campus of Haematology Franco e Piera Cutino- A.O.O.R. “Villa Sofia-Cervello”, Palermo, Italy
²2Dipartimento di Scienze Mediche, Sezione Talassemici Adulti, University of Cagliari, Cagliari, Italy
³Azienda Ospedaliera Univiversità Policlinico “Vittorio Emanuele -P.O. Ferrarotto, Catania, Italy
⁴Centro Microcitemia, A.O., Gela (CL), Italy
⁵A.O.O.C.R., Sciacca, Italy
⁶U.O. Genetica ed Immunologia Pediatrica, Policlinico “G. Martino”, Messina, Italy
⁷Unità Operativa Complessa Ematologia con Talassemia, ARNAS Civico, Palermo, Italy
⁸U.O.C. 7a Pediatria per le Emopatie Ereditarie, Centro di Prevenzione Diagnosi e Cura della Talassemia, A.O. “G. Di Cristina”, Palermo, Italy
⁹UOD Malattie rare del globulo rosso, AORN, Napoli, Italy
¹⁰Garibaldi Hospital, Catania, Italy
¹¹U.O.S. di Talassemia, Ospedale San Giovanni di Dio, Agrigento, Italy
¹²Ospedale Umberto I, Siracusa, Italy
¹³Section of Hematology, A.O. Sant'Elia, Caltanissetta, Italy
¹⁴A.O.‘‘M. Paternò Arezzo’’, Ragusa, Italy
¹⁵CMR Unit, Fondazione G. Monasterio CNR-Regione Toscana, Pisa, Italy

Veronica Agrigento^*, Sclafani Serena^*, Paolo Rigano^*, Rita Barone^*, Giuseppina Calvaruso^*, Rosario Di Maggio^*, Massimiliano Sacco^*, Lorella Pitrolo^*, Angela Vitrano^*, Aurelio Maggio and Elena D'Alcamo^*

Campus of Haematology Franco e Piera Cutino- A.O.O.R. “Villa Sofia-Cervello”, Palermo, Italy

Tamara K Moyo, MD, PhD¹, Anass Bouchnita, MS^2*, Nathalie Eymard, PhD^2*, Vitaly Volpert, PhD^2* and Mark J. Koury, MD¹

¹Division of Hematology/Oncology, Vanderbilt University, Nashville, TN
²Institut Camille Jordan, University Lyon 1, Villeurbanne, France