Elena Baiamonte^1*, Rita Barone^1*, Rosalia Di Stefano^1*, Melania Lo Iacono^1*, Barbara Spina^1*, Flavia Contino^2*, Rosario Di Maggio^1*, Massimiliano Sacco^1*, Angela Vitrano^1*, Salvatore Feo^2*, Aurelio Maggio¹ and Santina Acuto^1*

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 Zhu^1*, Kyung Chin^2*, Wulin Aerbajinai,^1*, Hongzhen Li^1*, Chutima Kumkhaek^2*, Matthew Hsieh² and Griffin P Rodgers²

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

Elena Baiamonte^1*, Rosalia Di Stefano^1*, Melania Lo Iacono^1*, Barbara Spina^1*, Angela Vitrano^1*, Rosario Di Maggio^1*, Massimiliano Sacco^1*, Giovanni Spinelli^2*, Aurelio Maggio¹, Santina Acuto^1* and Vincenzo Cavalieri^2,3*

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, BS^1,2*, David R Myers, PhD^1,2, Jordan E Shields, MS^2*, Byungwook Ahn, PhD^1,2*, Yongzhi Qiu, PhD^1,2*, Caroline Hansen, BS^2,3*, Yumiko Sakurai, MSc^1,2*, Robert Moot, BS^2*, H. Trent Spencer, PhD², Christopher B Doering, PhD⁴ and Wilbur A Lam, MD, PhD^1,2

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, PhD^1,2*, Kevin G Haworth, PhD^1*, Guy Sauvageau, M.D., Ph.D.³, Shelly Heimfeld, PhD¹, Jonah D Hocum, B.S.^4*, Grant D. Trobridge, PhD^5* and Hans-Peter Kiem, MD^1,2,6

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, PhD^1,2,3*, Christine Happle, MD^4*, Takuji Suzuki, MD^5*, Miriam Hetzel^1,2*, Kevin A. Link, PhD⁶, Adele Mucci^1,2*, Paritha Arumugam, PhD^7*, Jens Bankstahl, PhD^8*, Dagmar Dilloo, MD⁹, Punam Malik, MD^6*, Carolyn Lutzko, PhD^6*, Axel Schambach, MD, PhD^2*, Bruce Colston Trapnell, MD^5*, Gesine Hansen, MD^4* and Thomas Moritz, MD^1,2*

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, PhD^2*, Irene Zolotukhin, MS^3*, Katherine A. High, MD^4*, Arun Srivastava, PhD^2*, Ype P. de Jong, MD, PhD^5*, Koen Vercauteren, PhD^6* and Jing Xiao^6*

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, DVM^1,2*, Hideto Matsui, MD, PhD¹, Yasunori Matsunari, MD^1*, Sunao Ochi, DVM^1*, Midori Shima, MD, PhD³, Yasushi Fukuoka^4*, Takeshi Sato, MD^4*, Hiroshi Anai, MD, Ph.D^4*, Kimihiko Kichikawa, MD, Ph.D^4*, Masanari Nakayama, DVM, PhD^5*, Akitsu Hotta, Ph.D^6* and Mitsuhiko Sugimoto, MD, Ph.D^1*

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, PHD^1*, Qizhao Wang, PhD^2* and Weidong Xiao, PhD^3*

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.D^2*, Jordan E Shields, MS^3*, William Swaney^4*, Lilith Reeves^5*, 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, PhD^1,2*, Rajiv Sharma, PhD^2*, Yannick Doyon, PhD^3,4*, Thomas Wechsler, PhD^3*, David Paschon, PhD^3*, Robert J. Davidson^2*, Shangzhen Zhou, MD^2*, Philip D Gregory, PhD^3*, Michael C. Holmes, PhD^3* 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, MD^1,2*, Juan Chen, PhD^1*, Jocelyn Schroeder^1*, Christina K Baumgartner, PhD^1*, 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, PhD^1*, John M. Rossi, MS^1*, Yizhou Jiang, PhD^1*, Lynn Navale, MS^1*, Yueh-wei Shen, MS^1*, Marika Sherman, MS^1*, Armen Mardiros, PhD¹, Sean C. Yoder, MS^1*, William Y. Go, MD, PhD¹, Steven A. Rosenberg, MD, PhD^2*, Jeff Wiezorek, MD, MS^1*, David D. Chang, MD, PhD^1*, James N. Kochenderfer, MD³ and Margo R. Roberts, PhD^1*

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, MS^1*, Waleed Haso, MS^2*, Sang Minh Nguyen, BS^1* 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. Rufener^1*, Philip Olsen^1*, Sang Yun Lee, PhD^1*, Michael C Jensen, MD^1,2*, Ajay K. Gopal, MD¹, Barbara Pender, MS^1*, Damian J. Green, MD¹, David G. Maloney, MD, PhD¹, Stanley R. Riddell, MD^1*, 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, PharmD^1*, Lounes Djerroudi, MD^1*, Isabelle Hmitou, PhD^1*, Marina Cavazzana, MD, PhD^1,2,3 and Fabien Touzot, MD, PhD^1,3*

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 Patel^3*, 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, Master^1*, David M. Barrett, MD, PhD^2*, 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