-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. Gene Therapy and Transfer: Poster III

Gene Therapy and Transfer Program: Oral and Poster Abstracts
Type: Poster
Monday, December 7, 2015: 6:00 PM-8:00 PM
Hall A, Level 2 (Orange County Convention Center)

Elena Baiamonte1*, Rita Barone1*, Rosalia Di Stefano1*, Melania Lo Iacono1*, Barbara Spina1*, Flavia Contino2*, Rosario Di Maggio1*, Massimiliano Sacco1*, Angela Vitrano1*, Salvatore Feo2*, Aurelio Maggio1 and Santina Acuto1*

1Campus of Haematology Franco e Piera Cutino- A.O.O.R. “Villa Sofia-Cervello”, Palermo, Italy
2Dpt. of Biological, Chemical and Pharmaceutical Sciences and Technologies and Center for Human Health Advanced Biotechnologies - CHAB, University of Palermo, Palermo, Italy

Jianqiong Zhu1*, Kyung Chin2*, Wulin Aerbajinai,1*, Hongzhen Li1*, Chutima Kumkhaek2*, Matthew Hsieh2 and Griffin P Rodgers2

1Molecular and Clinical Hematology Branch, NHLBI/NIH, Bethesda
2Molecular and Clinical Hematology Branch, NHLBI/NIH, Bethesda, MD

Elena Baiamonte1*, Rosalia Di Stefano1*, Melania Lo Iacono1*, Barbara Spina1*, Angela Vitrano1*, Rosario Di Maggio1*, Massimiliano Sacco1*, Giovanni Spinelli2*, Aurelio Maggio1, Santina Acuto1* and Vincenzo Cavalieri2,3*

1Campus of Haematology Franco e Piera Cutino- A.O.O.R. “Villa Sofia-Cervello”, Palermo, Italy
2Department of Biological, Chemical and Pharmaceutical Sciences and Technologies (STEBICEF), University of Palermo, Palermo, Italy
3Mediterranean Center for Human Health Advanced Biotechnologies (CHAB), University of Palermo, Palermo, Italy

Reginald Tran, BS1,2*, David R Myers, PhD1,2, Jordan E Shields, MS2*, Byungwook Ahn, PhD1,2*, Yongzhi Qiu, PhD1,2*, Caroline Hansen, BS2,3*, Yumiko Sakurai, MSc1,2*, Robert Moot, BS2*, H. Trent Spencer, PhD2, Christopher B Doering, PhD4 and Wilbur A Lam, MD, PhD1,2

1Wallace H. Coulter Department of Biomedical Engineering, Georgia Institute of Technology and Emory University, Atlanta, GA
2Department of Pediatrics, Aflac Cancer Center and Blood Disorders Service of Children's Healthcare of Atlanta and Emory University School of Medicine, Atlanta, GA
3Georgia Institute of Technology, Atlanta, GA
4Aflac Cancer and Blood Disorders Center, Department of Pediatrics, Emory University School of Medicine, Atlanta, GA

Jennifer E Adair, PhD1,2*, Kevin G Haworth, PhD1*, Guy Sauvageau, M.D., Ph.D.3, Shelly Heimfeld, PhD1, Jonah D Hocum, B.S.4*, Grant D. Trobridge, PhD5* and Hans-Peter Kiem, MD1,2,6

1Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA
2University of Washington, Seattle, WA
3Department of Medicine, Faculty of Medicine, Université de Montréal, Montréal, QC, Canada
4Department of Pharmacy, Washington State University, Pullman, WA
5Washington State University, Pullman, WA
6Department of Pathology, University of Washington School of Medicine, Seattle

Nico Lachmann, PhD1,2,3*, Christine Happle, MD4*, Takuji Suzuki, MD5*, Miriam Hetzel1,2*, Kevin A. Link, PhD6, Adele Mucci1,2*, Paritha Arumugam, PhD7*, Jens Bankstahl, PhD8*, Dagmar Dilloo, MD9, Punam Malik, MD6*, Carolyn Lutzko, PhD6*, Axel Schambach, MD, PhD2*, Bruce Colston Trapnell, MD5*, Gesine Hansen, MD4* and Thomas Moritz, MD1,2*

1Reprogramming and Gene Therapy Group, REBIRTH Cluster of Excellence, Hannover Medical School, Hannover, Germany, Hannover, Germany
2Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany
3JRG Translational Hematology of Congenital Diseases, REBIRTH Cluster of Excellence, Hannover Medical School, Hannover, Germany
4Department of Pediatric Pneumology, Allergology and Neonatology, Hannover Medical School, Hannover, Germany
5Division of Pulmonary Biology, Children’s Hospital Medical Center, Cincinnati, OH
6Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
7Translational Pulmonary Center, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
8Institute for Preclinical Molecular Imaging, Hannover Medical School, Hannover, Germany, Hannover, Germany
9Department of Pediatric Hematology and Oncology, Center for Child and Adolescent Medicine, Rheinische Friedrich-Wilhelms University, Bonn, Germany, Dusseldorf, Germany

Roland W. Herzog, PhD1, Brad E. Hoffman, PhD2*, Irene Zolotukhin, MS3*, Katherine A. High, MD4*, Arun Srivastava, PhD2*, Ype P. de Jong, MD, PhD5*, Koen Vercauteren, PhD6* and Jing Xiao6*

1Pediatrics, University of Florida, Gainesville, FL
2Department of Pediatrics, University of Florida, Gainesville, FL
3Pediatrics/CMT, University of Florida, Gainesville, FL
4Spark Therapeutics, Inc., Philadelphia, PA
5Weill Cornell Medical College, Cornell University, New York, NY
6Laboratory of Virology and Infectious Disease, Rockefeller University, New York, NY

Masashi Noda, DVM1,2*, Hideto Matsui, MD, PhD1, Yasunori Matsunari, MD1*, Sunao Ochi, DVM1*, Midori Shima, MD, PhD3, Yasushi Fukuoka4*, Takeshi Sato, MD4*, Hiroshi Anai, MD, Ph.D4*, Kimihiko Kichikawa, MD, Ph.D4*, Masanari Nakayama, DVM, PhD5*, Akitsu Hotta, Ph.D6* and Mitsuhiko Sugimoto, MD, Ph.D1*

1Regulatory Medicine for Thrombosis, Nara Medical University, Kashihara, Japan
2Tennoji Veterinary Hospital, Osaka, Japan
3Pediatrics, Nara Medical University, Kashihara, Japan
4Radiology, Nara Medical University, Kashihara, Japan
5Nakayama Veterinary Hospital, Nara, Japan
6Reprogramming Science, Center for iPS Cell Research and Application, Kyoto University, Kyoto, Japan

Jenni A. Firrman, PHD1*, Qizhao Wang, PhD2* and Weidong Xiao, PhD3*

1Temple University School of Medicine, Philadelphia, PA
2Sol Sherry Thrombosis Research Center, Temple university, Philadelphia, PA
3Department of Microbiology and Immunology, Temple university, Philadelphia, PA

Christopher B Doering, PhD1, Gabriela Denning, Ph.D2*, Jordan E Shields, MS3*, William Swaney4*, Lilith Reeves5*, Punam Malik, MD6 and H. Trent Spencer, PhD3

1Aflac Cancer and Blood Disorders Center, Department of Pediatrics, Emory University School of Medicine, Atlanta, GA
2Expression Therapeutics, Tucker, GA
3Department of Pediatrics, Aflac Cancer Center and Blood Disorders Service of Children's Healthcare of Atlanta and Emory University School of Medicine, Atlanta, GA
4Cincinnati Children's Hospital Medical Center, cincinnati, OH
5Cincinnati Children's Hospital Medical Center, Cincinnati, OH
6Divisions of Hematology, Experimental Hematology and Cancer Biology, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, OH

Xavier M. Anguela, PhD1,2*, Rajiv Sharma, PhD2*, Yannick Doyon, PhD3,4*, Thomas Wechsler, PhD3*, David Paschon, PhD3*, Robert J. Davidson2*, Shangzhen Zhou, MD2*, Philip D Gregory, PhD3*, Michael C. Holmes, PhD3* and Katherine A. High, M.D.1,2,5

1Spark Therapeutics, Inc., Philadelphia, PA
2The Children's Hospital of Philadelphia, Philadelphia, PA
3Sangamo BioSciences, Richmond, CA
4Department of Molecular Medicine, Laval University, Centre Hospitalier Universitaire de Québec Research Center, Laval, QC, Canada
5Howard Hughes Medical Institute, Philadelphia, PA

Xiaofeng Luo, MD1,2*, Juan Chen, PhD1*, Jocelyn Schroeder1*, Christina K Baumgartner, PhD1*, Jianda Hu, MD, PhD3 and Qizhen Shi, MD, PhD1

1Medical College of Wisconsin, Blood Research Institute, Children's Research Institute, Milwaukee, WI
2Department of Hematology, Union Hospital, Fujian Medical University, Fuzhou, China
3Fujian Institute of Hematology, Fujian Medical University Union Hospital, Fuzhou, China

Reona Sakemura, MD*, Seitaro Terakura, MD, PhD*, Keisuke Watanabe, MD, PhD*, Kotaro Miyao, MD*, Daisuke Koyama, MD*, Tatsunori Goto, MD*, Ryo Hanajiri, MD*, Tetsuya Nishida, MD, PhD*, Makoto Murata, MD, PhD and Hitoshi Kiyoi, MD, PhD

Department of Hematology and Oncology, Nagoya University Graduate School of Medicine, Nagoya, Japan

Rayne H. Rouce, MD, Kristen Fousek*, Nabil Ahmed, MD*, Stephen Gottschalk, MD, Barbara Savoldo, MD*, Gianpietro Dotti, MD*, Carlos A. Ramos, MD, Chrystal Louis, MD*, Cliona M. Rooney, PhD*, Malcolm K. Brenner, MD, PhD and Helen E. Heslop, MD

Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital and Houston Methodist Hospital, Houston, TX

Adrian Bot, MD, PhD1*, John M. Rossi, MS1*, Yizhou Jiang, PhD1*, Lynn Navale, MS1*, Yueh-wei Shen, MS1*, Marika Sherman, MS1*, Armen Mardiros, PhD1, Sean C. Yoder, MS1*, William Y. Go, MD, PhD1, Steven A. Rosenberg, MD, PhD2*, Jeff Wiezorek, MD, MS1*, David D. Chang, MD, PhD1*, James N. Kochenderfer, MD3 and Margo R. Roberts, PhD1*

1Kite Pharma, Santa Monica, CA
2Surgery Branch, NCI, NIH, Bethesda, MD
3Experimental Transplantation and Immunology Branch, National Cancer Institute/National Institutes of Health, Bethesda, MD

Haiying Qin, MS1*, Waleed Haso, MS2*, Sang Minh Nguyen, BS1* and Terry J Fry, M.D.3

1Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD
2National Cancer Institute, National Institutes of Health, Bethesda, MD
3Pediatric Oncology Branch, Children's National Medical Center, Washington, DC

Gregory A. Rufener1*, Philip Olsen1*, Sang Yun Lee, PhD1*, Michael C Jensen, MD1,2*, Ajay K. Gopal, MD1, Barbara Pender, MS1*, Damian J. Green, MD1, David G. Maloney, MD, PhD1, Stanley R. Riddell, MD1*, Oliver W Press, MD, PhD1 and Brian G. Till, MD1

1Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA
2Department of Pediatrics, University of Washington, Seattle, WA

Amani Ouedrani, PharmD1*, Lounes Djerroudi, MD1*, Isabelle Hmitou, PhD1*, Marina Cavazzana, MD, PhD1,2,3 and Fabien Touzot, MD, PhD1,3*

1Biotherapy Department, Hôpital Universitaire Necker -Enfants Malades, Paris, France
2INSERM-APHP, CIC Biothérapie GHU Ouest, Paris, France
3IMAGINE Institute, Université Paris Descartes, Sorbonne Paris Cité, Paris, France

Laurel Christine Truscott, MD1, Sarah Larson, MD2, Amie Patel3*, Roy L. Kao, MD4 and Satiro N. De Oliveira, MD4

1Ucla-Mattel Children's Hospital, Los Angeles, CA
2University of California, Los Angeles, CA
3Western University of Health Sciences, Pomona, CA
4Dept. of Pediatrics, University of California, Los Angeles, Los Angeles, CA

Xiaojun Liu, Master1*, David M. Barrett, MD, PhD2*, Shuguang Jiang, Ph.D.1*, Chongyun Fang, Ph.D.1*, Stephan A. Grupp, MD, PhD2, Carl H. June, MD1 and Yangbing Zhao, Ph.D. M.D.3*

1Center for Cellular Immunotherapies, University of Pennsylvania, Philadelphia, PA
2Division of Oncology and Center for Childhood Cancer Research, Children's Hospital of Philadelphia, Philadelphia, PA
3Center for Cellular Immunotherapies, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA

*signifies non-member of ASH