-Author name in bold denotes the presenting author
-Asterisk * with author name denotes a Non-ASH member
Clinically Relevant Abstract denotes an abstract that is clinically relevant.

PhD Trainee denotes that this is a recommended PHD Trainee Session.

Ticketed Session denotes that this is a ticketed session.

112.O1.6 112. Thalassemia and Globin Gene Regulation: Molecular Mechanisms of Thalassemia

Thalassemia and Globin Gene Regulation Program: Oral and Poster Abstracts
Type: Oral
Monday, December 5, 2016: 4:30 PM-6:00 PM
Room 7AB (San Diego Convention Center)
Moderators:
Olivier Hermine, MD, PhD, Hôpital Universitaire Necker-Enfants Malades and Vip Viprakasit, MD, PhD, Siriraj Hospital, Madihol University
Disclosures:
Hermine: Alexion: Research Funding; Novartis: Research Funding; Celgene: Research Funding; AB science: Consultancy, Equity Ownership, Membership on an entity's Board of Directors or advisory committees, Patents & Royalties, Research Funding, Speakers Bureau. Viprakasit: Novartis: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; Agios: Consultancy, Honoraria; Celgene: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Mergansers: Consultancy, Membership on an entity's Board of Directors or advisory committees; Roche Diagnostics: Consultancy, Honoraria, Research Funding; Sebia: Honoraria, Research Funding.
4:30 PM

Jaira F. de Vasconcellos1*, Y. Terry Lee1*, Colleen Byrnes1*, Benjamin A. Clarke2*, Pauline C. Xu1*, Antoinette Rabel1*, Richard L. Proia2* and Jeffery L. Miller1

1Molecular Genomics and Therapeutics Section, Genetics of Development and Disease Branch, National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD
2Genetic Disease Research Section, Genetics of Development and Disease Branch, National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD

4:45 PM

Carina Levin, MD1,2*, Annie Rebibo-Sabbah3*, Ariel Koren, MD1,2*, Benjamin Brenner, MD1,4 and Anat Aharon, PhD1,3*

1The Bruce Rappaport Faculty of Medicine, Technion – Israel Institute of Technology, Haifa, Israel
2Pediatric Hematology Unit, Emek Medical Center, Afula, Israel
3Department of Hematology and Bone Marrow Transplantation, Rambam Health Care Campus, Haifa, Israel
4Thrombosis and Hemostasis Unit, Rambam Health Care Campus, Haifa, Israel

5:00 PM

Maria Feola, BSc, MSc1,2*, Andrea Zamperone, PhD3*, Weili Bao, MS4*, Tenzin Choesang, MS4*, Huihui Li, MS5,6*, Guiyuan Li, MD5*, Shilpa M. Hattangadi, MD7, Christopher E. Mason, PhD8*, Peng Ji, MD, PhD9, Antonia Follenzi, MD PhD10* and Yelena Ginzburg11*

1Division of Hematology and Medical Oncology, Icahn School of Medicine at Mount Sinai / The Tisch Cancer Institute, New York, NY
2Department of Health Sciences, University of Piemonte Orientale "Amedeo Avogadro", NOVARA, Italy
3Albert Einstein College of Medicine, Bronx, NY
4New York Blood Center, New York, NY
5Cancer Research Institute, Central South University, Changsha, China
6Icahn School of Medicine at Mount Sinai / The Tisch Cancer Institute, New York, NY
7Fegan 7, Boston, MA
8Institute for Computational Biomedicine and Department of Physiology and Biophysics, Weill Cornell Medical College, New York, NY
9Pathology, Northwestern University Medical School, Chicago, IL
10University of Piemonte Orientale, Amedeo Avogadro, Novara, Italy
11Icahn School of Medicine at Mount Sinai, New York

5:15 PM

Carla Casu1*, Paraskevi Rea Oikonomidou, M.D.2*, Vania Lo Presti1*, Mariam Aghajan, PhD3*, Shuling Guo, PhD3*, Abdulmalik Osheiza1*, Luca Melchiori4*, Pedro Ramos5* and Stefano Rivella, PhD2

1Children’s Hospital of Philadelphia (CHoP), Philadelphia, PA
2Children's Hospital of Philadelphia, Philadelphia, PA
3Ionis Pharmaceuticals, Inc., Carlsbad, CA
4Adaptimmune Ltd, Abingdon, United Kingdom
5Novartis, Basel, Switzerland

5:30 PM

Mariam Aghajan, PhD1*, Carla Casu, PhD2*, Vania lo Presti2*, Sheri Booten1*, Brett P. Monia, PhD1, Stefano Rivella, PhD2 and Shuling Guo, PhD1*

1Ionis Pharmaceuticals, Inc., Carlsbad, CA
2Children's Hospital of Philadelphia, Philadelphia, PA

5:45 PM

Alisa Cheung Dong1,2*, Valentina Ghiaccio1*, Irene Motta, MD3*, Shuling Guo, PhD4*, Raechel Peralta, MS4*, Coralea Stephanou, PhD5*, Paola Delbini, PhD6*, Connie Chen7*, Soteroula Christou8*, Marina Kleanthous, PhD9*, Maria Domenica Cappellini, MD10, Osheiza Abdulmalik, DVM, MS11, Laura Breda, PhD12* and Stefano Rivella, PhD12

1Hematology, Children's Hospital of Philadelphia, Philadelphia, PA
2Graduate School of Medical Sciences, Weill Cornell Medical College of Cornell University, New York, NY
3Rare Diseases Center, Department of Medicine and Medical Specialities, Ca’ Granda IRCCS Foundation Ospedale Maggiore Policlinico, Milan, Italy
4Ionis Pharmaceuticals, Inc., Carlsbad, CA
5Molecular Genetics of Thalassaemia Department, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus
6Rare Diseases Center, Department of Medicine and Medical Specialities, Ca’ Granda IRCCS Foundation Ospedale Maggiore Policlinico, Milan, Italy
7Weill Cornell Medical College, New York
8Thalassaemia Center, Archbishop Makarios III Hospital, Nicosia, Cyprus
9The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus
10Department of Medicine and Medical Specialities, Department of Clinical Science and Community Health, Rare Diseases Center, Fondazione IRCCS “Ca’ Granda”, Ospedale Maggiore Policlinico, University of Milan, Milan, Italy
11Division of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA
12Children's Hospital of Philadelphia, Philadelphia, PA

*signifies non-member of ASH