-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.

801.O2.6 801. Gene Therapy and Transfer: Gene Therapy for Immune Disorders and Cancer

Gene Therapy and Transfer Program: Oral and Poster Abstracts
Type: Oral
Sunday, December 6, 2015: 12:00 PM-1:30 PM
W230, Level 2 (Orange County Convention Center)
Moderators:
Isabelle Riviere, PhD, Memorial Sloan Kettering Cancer Center and Saar I Gill, MBBS, PhD, University of Pennsylvania

Disclosures:
Riviere: Juno Therapeutics: Other: Co-founder, stockholder and consultant . Gill: Novartis: Patents & Royalties , Research Funding .
12:00 PM

Francesca Ferrua, MD1,2,3*, Maria Pia Cicalese, MD, PhD1,3*, Stefania Galimberti, PhD4*, Samantha Scaramuzza, PhD1*, Stefania Giannelli, PhD1*, Roberta Pajno, MD2,3*, Francesca Dionisio, PhD1*, Luca Biasco, PhD1*, Maria Carmina Castiello, PhD1*, Miriam Casiraghi, RN1,3*, Marcella Facchini, PhD1*, Andrea Finocchi, MD5*, Ayse Metin, MD, PhD6*, Jordan S. Orange, MD, PhD7*, Michael H. Albert, MD8*, Carmen Petrescu, MD9*, Marita Bosticardo, PhD1*, Anna Villa, MD1,10*, Chris Dott, PhD11*, Koen van Rossem, MD, MSc, PhD12*, Maria Grazia Valsecchi, PhD4, Fabio Ciceri, MD13*, Maria Grazia Roncarolo, MD1,2,14, Luigi Naldini, MD, PhD1,2* and Alessandro Aiuti, MD, PhD1,2,3

1San Raffaele Telethon Institute for Gene Therapy (TIGET), San Raffaele Scientific Institute, Milan, Italy
2Vita-Salute San Raffaele University, Milan, Italy
3Pediatric Immunohematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Milan, Italy
4Center of Biostatistics for Clinical Epidemiology, Department of Health Sciences, University of Milano-Bicocca, Monza, Italy
5DPUO, University Department of Pediatrics, Bambino Gesù Children’s Hospital and Tor Vergata University, School of Medicine, Rome, Italy
6Pediatric Immunology and Allergy Department, Ankara Children's Hematology Oncology Training and Research Hospital, Ankara, Turkey
7Center for Human Immunobiology, Texas Children's Hospital and Baylor College of Medicine, Houston, TX
8Dr von Hauner Children's Hospital, Ludwig Maximillian’s University, Munich, Germany
9Department of Public Health, School of Medicine, V. Babeş University of Medicine & Pharmacy, Timişoara, Romania
10Milan Unit, Istituto di Ricerca Genetica e Biomedica, Consiglio Nazionale delle Ricerche, Milan, Italy
11CSD Pharma Consulting Ltd., London, United Kingdom
12Rare Diseases Unit, GlaxoSmithKline, Brentford, United Kingdom
13Hematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Milan, Italy
14Division of Stem Cell Transplantation and Regenerative Medicine and iSCBRM, Department of Pediatrics, Stanford University, Stanford, CA

12:15 PM

Julia I Chu, MD, MPH1,2, Lauren A Henderson, MD3*, Myriam Armant, PhD2*, Frances Male, BA4*, Colleen H Dansereau, MSN, RN5*, Brenda MacKinnon, RN6*, Christopher J Burke1*, Matthew E Cavanaugh1*, Wendy B. London, PhD1,2*, Isil B Barlan, MD7*, Ahmet Özen, MD7*, Safa Baris, MD7*, Elif Karakoc-Aydiner, MD7*, Ayça Kiykim, MD7*, Imelda Celine Hanson, MD8*, Jenny M. Despotovic, DO, MS9, Akihiko Saitoh, MD, PhD10*, Takayuki Takachi, MD, PhD10*, Kohsuke Imai, MD, PhD11*, Alejandra King, MD12*, Silvia Arredondo, MD13*, Jerome Ritz, MD14, Frederic D Bushman, PhD4*, Anne Galy, PharmD, PhD15, Luigi Daniele Notarangelo, MD3*, David A. Williams, MD1,2 and Sung-Yun Pai, MD1,2

1Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA
2Division of Hematology-Oncology, Boston Children's Hospital, Boston, MA
3Division of Immunology, Boston Children's Hospital, Boston, MA
4Department of Microbiology, University of Pennsylvania School of Medicine, Philadelphia, PA
5Division of Pediatric Hematology, Boston Children's Hospital, Boston, MA
6Division of Pediatric Hematology, Boston Children’s Hospital, Boston, MA
7Division of Pediatric Allergy/Immunology, Marmara University, Istanbul, Turkey
8Division of Pediatric Immunology, Allergy and Rheumatology, Texas Children's Hospital, Houston, TX
9Texas Children's Hematology Center, Baylor College of Medicine, Houston, TX
10Department of Pediatrics, Niigata University Graduate School of Medical and Dental Sciences, Niigata, Japan
11Department of Pediatrics and Developmental Biology, Tokyo Medical and Dental University, Tokyo, Japan
12Hospital Luis Calvo Mackenna, Santiago, Chile
13Pediatric Service, Hospital Regional Talca, Talca, Chile
14Division of Hematologic Malignancies, Dana-Farber Cancer Institute, Boston, MA
15Genethon, Evry, France

12:30 PM

Suk See De Ravin, MD PhD1*, Xiaolin Wu2*, Narda Theobald3*, Janet S Lee4*, John Gray, Ph.D.5*, Imelda Celine Hanson, MD6*, Luigi Notarangelo, MD PhD7*, Brian P. Sorrentino, MD8,9 and Harry L. Malech, MD10

1LHD, National Institutes of Health, Bethesda
2Cancer Reserach Technology Program, Leidos Biomedical Reserach, Inc., Frederick
3LHD, NIAID, NIH, Bethesda, MD
4NIH, Bethesda
5Research & Developement, Audentes Therapeutics, San Francisco, CA
6Division of Pediatric Immunology, Allergy and Rheumatology, Texas Children's Hospital, Houston, TX
7Harvard Medical School, Boston
8Division of Experimental Hematology, St. Jude Children's Research Hospital, Memphis, TN
9Hematology, St. Jude Children's Research Hospital, Memphis, TN
10Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, Bethesda, MD

12:45 PM

Olivier Humbert, PhD1*, Christopher R Burtner, PhD1*, Patricia O'Donnell2*, Daniel R Humphrys, B.S.1*, Nicholas Hubbard3*, Frieda Chan, PhD1*, Jennifer E Adair, PhD1,4*, Grant D. Trobridge, PhD5*, Troy Torgerson, MD, PhD3,4*, Andrew M. Scharenberg, MD4*, David J. Rawlings, MD3,4*, Peter Felsburg2* and Hans-Peter Kiem, MD1,4

1Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA
2University of Pennsylvania, philadelphia, PA
3Seattle Children's Research Institute, Seattle, WA
4University of Washington, Seattle, WA
5Washington State University, Pullman, WA

1:00 PM

Giacomo Oliveira, PhD1,2*, Eliana Ruggiero, PhD3,4*, Maria Teresa Lupo Stanghellini, MD5*, Nicoletta Cieri, MD, PhD3*, Mattia D'Agostino, MD3*, Raffaele Fronza, PhD4*, Christina Lulay4*, Francesca Dionisio, PhD6*, Sara Mastaglio, MD3,5*, Raffaella Greco, MD7*, Jacopo Peccatori, MD5*, Alessandro Aiuti, MD8, Luca Biasco, PhD6*, Attilio Bondanza, MD, PhD9, Antonio Lambiase, MD10*, Catia Traversari, PhD10*, Luca Vago, MD, PhD7,11, Christof von Kalle, MD4*, Manfred Schmidt, PhD4*, Claudio Bordignon, MD1,10, Fabio Ciceri, MD12* and Chiara Bonini, MD13

1Vita-Salute San Raffaele University, Milan, Italy
2Experimental Hematology Unit, San Raffaele Scientific Institute, Milano, Italy
3Experimental Hematology Unit, San Raffaele Scientific Institute, Milan, Italy
4Department of Translational Oncology, National Center for Tumor Diseases and German Cancer Research Center, Heidelberg, Germany
5Hematology and Bone Marrow Transplantation Unit, San Raffaele Scientific Institute, Milan, Italy
6San Raffaele Telethon Institute for Gene Therapy (TIGET), San Raffaele Scientific Institute, Milan, Italy
7Unit of Hematology and Bone Marrow Transplantation, San Raffaele Scientific Institute, Milano, Italy
8Telethon Institute for Gene Therapy (HSR-TIGET), San Raffaele Scientific Institute, Milan, Italy
9Innovative Immunotherapies Unit, San Raffaele Hospital Scientific Institute, Milan, Italy
10MolMed S.p.A., Milan, Italy
11Unit of Immunogenetics, Leukemia Genomics and Immunobiology, San Raffaele Scientific Institute, Milano, Italy
12Hematology and Bone Marrow Transplantation Unit, San Raffaele Hospital Scientific Institute, Milan, Italy
13Experimental Hematology Unit, San Raffaele Hospital Scientific Institute, Milan, Italy

1:15 PM

Lung-Ji Chang, PhD1,2, Lujia Dong, M.D.3, Jun Zhu, MD4, Zhitao Ying, MD4*, Hao-Hsian Kuo, MSc1,3*, Yuchen Liu, MSc1,3*, Yu-Qin Song, M.D.4*, Xiao-Pei Wang, M.D.4*, Yong-Qian Jia, M.D.5*, Ting Niu, MD, PhD5, Ting Liu, MD, PhD5, Ling Pan, M.D.5*, Zhuo-Gang Liu, M.D.6*, Tong Li, M.D.6*, Ying-Chun Li, M.D.7*, Kun Yao, MD7*, Xiaoyan Ke, MD, PhD7*, Hongmei Jing, M.D.8* and Fang Bao, M.D.8*

1Shenzhen Genoimmune Medical Institute (GIMI), Shenzhen, China
2Department of Molecular Genetics and Microbiology, University of Florida, Gainesville, FL
3America Yuva Biomed, Beijing, China
4Peking University Cancer Hospital, Beijing, China
5Department of Hematology, West China Hospital, Sichuan University, Chengdu, China
6Department of Hematology, Sheng-Jing Hospital of China Medical University, Shen-Yang, China
7Department of Hematology, Third Affiliated Hospital of Peking University, Beijing, China
8Department of Hematology, Peking University Third Hospital, Beijing, China

*signifies non-member of ASH