Santhosh Dhanraj, MSc^1,2*, Sethu Madhava Rao Gunja, MSc^3*, Adam P Deveau, BScH, MSc^4*, Mikael Nissbeck, MSc^3*, Boonchai Boonyawat, MD^2*, Andrew J Coombs, BScH^4*, Alessandra Renieri, MD^5*, Mafalda Mucciolo, MD^5*, Annabella Marozza, MD^5*, Sabrina Buoni^6*, Lesley Turner, MD^7*, Hongbing Li, PhD^2*, Ameer Jarrar, MSc^4*, Mathura Sabanayagam, BSc^2*, Melanie Kirby, MD^8*, Mary Shago, PhD⁹, Dalila Pinto, PhD^10*, Jason N. Berman, MD¹¹, Stephen W. Scherer, PhD, FRSC^2*, Anders Virtanen, PhD^3* and Yigal Dror, MD, FRCP(C)^1,2,12

¹Institute of Medical Science, University of Toronto, Toronto, ON, Canada
²Genetics and Genome Biology Program, The Hospital for Sick Children, Toronto, ON, Canada
³Department of Cell and Molecular Biology, Uppsala University, Uppsala, Sweden
⁴Department of Pediatrics, IWK Health Centre, Halifax, NS, Canada
⁵Medical Genetics, University of Siena, Siena, Italy
⁶Pediatric Neurology, University of Siena, Siena, Italy
⁷Discipline of Genetics, Memorial University of Newfoundland, St. John's, NF, Canada
⁸Division of Hematology/Oncology, Department of Pediatrics, The Hospital for Sick Children, Toronto, ON, Canada
⁹Cytogenetics Laboratory, Department of Paediatric Laboratory Medicine, The Hospital for Sick Children, Toronto, ON, Canada
¹⁰Departments of Psychiatry, and Genetics and Genomic Sciences, Seaver Autism Center, The Mindich Child Health & Development Institute, Mount Sinai School of Medicine, New York, NY
¹¹Pediatrics/Microbiology and Immunology, IWK Health Centre/Dalhousie University, Halifax, NS, Canada
¹²Marrow Failure and Myelodysplasia Program, Division of Hematology/Oncology, Department of Pediatrics, The Hospital for Sick Children, Toronto, ON, Canada

Wei Shi, Ph.D.^1*, Therese Vu, BSc^1*, Glen Boyle, Ph.D.^1*, Fares Al-Ejeh, Ph.D.^1*, Tej Pandita, Ph.D.^2*, Krzysztof Ginalski, Ph.D.^3*, Maga Rowicka, Ph.D.^4*, Steven W. Lane, MD^1,5,6 and Kum Kum Khanna, Ph.D.^1*

¹QIMR Berghofer Medical Research Institute, Brisbane, Australia
²The Houston Methodist Research Institute, Houston, TX
³University of Warsaw, Warsaw, Poland
⁴University of Texas Medical Branch, Galveston, TX
⁵Royal Brisbane and Women’s Hospital, Brisbane, Australia
⁶University of Queensland, Brisbane, Australia

Katie A Matatall, PhD^1*, Mira Jeong, Ph.D.², Sun Deqiang, PhD^2*, Claudine Salire^1* and Katherine Y. King, MD, PhD^1,2

¹Pediatric Infectious Diseases, Baylor College of Medicine, Houston, TX
²Center for Cell and Gene Therapy, Baylor College of Medicine, Houston, TX

Patali S. Cheruku, B.Sc.^1*, Ayla Cash, B.Sc.^1*, Cynthia E. Dunbar, M.D.¹, Neal S. Young, MD² and Andre Larochelle, MD, PhD²

¹National Heart, Lung and Blood Institute, Hematology Branch, National Institutes of Health, Bethesda, MD
²Hematology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD

Anne-Laure Grignon, MD, BA, MA¹, Daria V. Babushok, MD, PhD^2,3, Li Yimei, phD^4*, Helge Hartung, MD⁵, Ho-Sun Lam^6*, Monica Bessler, MD, PhD⁷ and Timothy S. Olson, MD PhD^8,9,10

¹Children's Hospital of Philadelphia, Philadelphia, PA
²Division of Hematology, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA
³Abramson Cancer Center, Department of Hematology/Oncology, Hospital of the University of Pennsylvania, Philadelphia, PA
⁴Perelman School of Medicine at the University of Pennsylvania, Department of Biostatistics and Epidemiology, Philadelphia, PA
⁵Department of Hematology/Oncology, Children's Hospital of Philadelphia, Philadelphia, PA
⁶Bone Marrow Failure Center, Children's Hospital of Philadelphia, Philadelphia
⁷Pediatrics, The Children's Hospital of Philadelphia, Philadelphia, PA
⁸Division of Oncology, Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, PA
⁹Comprehensive Bone Marrow Failure Center, The Children's Hospital of Philadelphia, Philadelphia, PA
¹⁰Comprehensive Bone Marrow Failure Center, Division of Hematology, Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, PA

Lucia Gargiulo, PhD^1*, Yoshitaka Zaimoku, MD^2*, Barbara Scappini, MD^3*, Hiroyuki Maruyama, MD^2*, Rie Ohumi^2*, Lucio Luzzatto, MD⁴, Shinji Nakao, MD, PhD² and Rosario Notaro, MD^1*

¹Core Research Laboratory, Istituto Toscano Tumori, Florence, Italy
²Cellular Transplantation Biology, Kanazawa University Graduate School of Medical Science, Kanazawa, Japan
³Hematology, Azienda Ospedaliero-Universitaria Careggi, Florence, Italy
⁴Istituto Toscano Tumori, Florence, Italy

Carlo Dufour, MD¹, Silvia Ravera, PhD^2*, Simone Cesaro, MD^3*, Roberta Bottega, PhD^4*, Cesare Usai, PhD^5*, Cipolli Marco, MD^6*, Anna Savoia^7*, Paolo Degan, PhD^8*, Michela Faleschini^9*, Paola Cuccarolo^10*, Marta Columbaro^11*, Fabio Corsolini^12* and Enrico Cappelli, PhD^13*

¹Istituto Giannina Gaslini, Genoa, Italy
²Università di Genova, Genova, Italy
³Azienda Ospedaliera Universitaria Integrata Verona, Verona, Italy
⁴I.R.C.C.S. "Burlo Garofolo", Trieste, Italy
⁵CNR, genova, Italy
⁶Azienda Ospedaliera Universitaria Integrata, Verona, Italy
⁷IRCCS Burlo Garofolo, Trieste, Italy
⁸IRCCS AOU San Martino - IST, Genova, Italy
⁹I.R.C.C.S., Trieste, Italy
¹⁰IRCCS AOU San Martino - IST, genova, Italy
¹¹Istituto Ortopedico Rizzoli, Bologna, Italy
¹²Ospedale G. Gaslini, genova, Italy
¹³Ospedale G. Gaslini, Genova, Italy

Youngme Yoon, B.S.^1*, Ashley N. Kamimae-Lanning, Ph.D.^2*, Kelsie Storm, MD², Natalya A Goloviznina, B.S.^3* and Peter Kurre, MD⁴

¹Pediatrics, Oregon Health & Science University, Portland, OR
²Papé Family Pediatric Research Institute, Oregon Stem Cell Center, Oregon Health & Science University, Portland, OR
³University of Minnesota, Minneapolis, MN
⁴Pediatrics Papé Family Pediatric Research Institute; Knight Cancer Institute; Cell & Developmental Biology, Oregon Health & Science University, Portland, OR

Erik Westin, PhD^1*, Larisa Pereboeva, MD, PhD², Divya Devadasan, MS^1*, Tim M. Townes, PhD³ and Frederick D Goldman, MD⁴

¹University of Alabama at Birmingham, Birmingham, AL
²Univ. of Alabama At Birmingham, Birmingham, AL
³Department of Biochemistry and Molecular Genetics, UAB Stem Cell Institute, University of Alabama at Birmingham, Birmingham, AL
⁴Department of Pediatrics, Division of Hematology Oncology, Children's Hospital of Alabama, University of Alabama at Birmingham, Birmingham, AL

Anita Hill^1*, Jorg Taubel^2*, Jim Bush^3*, Anna Borodovsky^4*, Noriyuki Kawahata^4*, Helen Mclean^4*, Christine Powell^4*, Prasoon Chaturvedi^5*, Garvin Warner^4*, Pushkal Garg^4* and Benny Sorensen⁴

¹St James' Institute of Oncology; Leeds Teaching Hospitals, Leeds, United Kingdom
²St George's University of London, London, United Kingdom
³Covance Clinical Research Unit, Leeds, United Kingdom
⁴Alnylam Pharmaceuticals, Cambridge, MA
⁵Alnylam Pharmaceutical, Cambridge, MA

Ayodele Alaiya, MPH, MBBS, PhD, FRCPath^1*, Hazza A Alzahrani, MD^2*, Zakia Shinwari, MSc^3*, Tarek Owaidah, MD, FRCPA⁴, Fahad Al Mohareb, MD^5*, Fahad Al Sharif, MD^6*, Randa Al Nounou, MD^7*, Naeem A. Chaudhri, MD, FACP², Ghuzayel Aldawsari, MD^2*, Amr Hanbali, MD^8*, Marwan Shaheen, MD^2*, Feras Alfraih, MD^2*, Syed Osman Ahmed, MD, MRCP, FRCPath^8* and Mahmoud Aljurf, MD, MPH²

¹Proteomics Unit, Stem Cell & Tissue Re-Engineering Program, King Faisal Specialist Hospital & Research Center, Riyadh, Saudi Arabia
²King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia
³Proteomics Unit, Stem cell and Tissue Re-Engineering Program, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia
⁴Pathology and Laboratory Medicine, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia
⁵Oncology Center, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia
⁶King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia
⁷Department of Pathology and Laboratory Medicine, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia
⁸Oncology Center, King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia

Luis Espinoza, MD, PhD^1*, Yoshinori Yoshida, MD, PhD^2*, Kazuhisa Chonabayashi, MD, PhD^3*, Takamasa Katagiri, PhD^4*, Yoshitaka Zaimoku, MD^5* and Shinji Nakao, MD, PhD⁵

¹Kanazawa University, Kanazawa, Japan
²Center for iPS cells Research and Application, Kyoto University, Kyoto, Japan
³Department of Reprogramming Science, Center for iPS Cell Research and Application, Kyoto University, Kyoto, Japan
⁴Graduate School of Medical Science, Clinical Laboratory Sciences, Kanazawa University, Kanazawa, Japan
⁵Cellular Transplantation Biology, Kanazawa University Graduate School of Medical Science, Kanazawa, Japan

Nicolas Waespe, MD^1,2, Santhosh Dhanraj, BSc, MSc^1,3*, Manju Wahala, MD^1*, Tom Enbar^1*, Bozana Zlateska^1*, Hongbing Li, PhD^1*, Robert J. Klaassen, MD, FRCP⁴, Conrad Fernandez, MD⁵, Rochelle Yanofsky, MD, FRCP(C)^6*, John K. Wu, MBBS, MSc, FRCPC⁷, Yves D. Pastore, MD⁸, Mariana Silva, MD, FRCPC^9*, Jeffrey H. Lipton, MD, PhD¹⁰, Josee Brossard, MD^11*, Bruno Michon, MD, FRCPC^12*, Sharon Abish, MD¹³, MacGregor Steele, MD, FRCP(C)¹⁴, Roona Sinha, MD^15*, Mark J. Belletrutti, MD, MSc¹⁶, Vicky R. Breakey, BSc, MD, MEd, FRCPC¹⁷, Lawrence Jardine, MD, BA, FRCPC¹⁸, Lisa Goodyear, MD, FRCPC^19*, Liat Kofler^1*, Ibrahim A. Ghemlas, MD^1,20*, Michaela Cada, MD, FRCPC, FAAP, MPH², Lillian Sung, MD, PhD²¹, Mary Shago, PhD²², Stephen W. Scherer, PhD, FRSC^1* and Yigal Dror, MD, FRCP(C)^1,2,23

¹Genetics and Genome Biology Program, The Hospital for Sick Children, Toronto, ON, Canada
²Marrow Failure and Myelodysplasia Program, Division of Hematology/Oncology, Department of Pediatrics, The Hospital for Sick Children, Toronto, ON, Canada
³Institute of Medical Sciences, University of Toronto, Toronto, ON, Canada
⁴Department of Pediatrics, Children's Hospital of Eastern Ontario, Ottawa, ON, Canada
⁵Pediatric Hematology/Oncology, IWK Health Centre, Halifax, NS, Canada
⁶Pediatric Hematology/Oncology, University of Manitoba, CancerCare Manitoba, Winnipeg, MB, Canada
⁷Division of Hematology/Oncology, UBC & B.C. Children's Hospital, Vancouver, BC, Canada
⁸CHU Sainte-Justine, Montreal, QC, Canada
⁹Kingston General Hospital, Kingston, ON, Canada
¹⁰Allogeneic Blood and Marrow Transplant Program, Princess Margaret Cancer Centre, University of Toronto, Toronto, ON, Canada
¹¹Centre hospitalier universitaire, Sherbrooke, QC, Canada
¹²Centre Hospitalier Universitaire de Quebec, Sainte-Foy, QC, Canada
¹³Pediatric Hematology Oncology, Montreal Children's Hospital, Montreal, QC, Canada
¹⁴Alberta Children's Hospital, Calgary, Canada
¹⁵Royal University Hospital, Saskatoon, SK, Canada
¹⁶Pediatrics, University of Alberta, Edmonton, AB, Canada
¹⁷Department of Pediatrics, McMaster University, Hamilton, ON, Canada
¹⁸Children's Hospital, London Health Sciences Centre, London, ON, Canada
¹⁹Pediatric Hematology/Oncology, Janeway Child Health Centre, St. John's, NF, Canada
²⁰Department of Pediatric Hematology/Oncology, King Faisal Specialist Hospital & Research Center, Riyadh, Saudi Arabia
²¹Population Health Sciences, Research Institute, Division of Hematology/Oncology, Department of Pediatrics, The Hospital for Sick Children, Toronto, ON, Canada
²²Cytogenetics Laboratory, Department of Paediatric Laboratory Medicine, The Hospital for Sick Children, Toronto, ON, Canada
²³Institute of Medical Science, University of Toronto, Toronto, ON, Canada

Racheli Ofir, PhD^1*, Lena Pinzur^2*, Akyüz Levent^3*, Zami Aberman^2*, Raphael Gorodetsky, PhD^4* and Hans Dieter Volk, PhD^3*

¹Pluristem Therapeutics Inc. - Haifa, Israel, HAIFA, Israel
²Pluristem Therapeutics, Haifa, Israel
³Charité - Universitätsmedizin Berlin, Berlin, Germany
⁴Hadassah - Hebrew University Medical Center, Jerusalem, Israel, HAIFA, Israel

Noriharu Nakagawa, MD^*, Hiroyuki Maruyama, MD^*, Yoshitaka Zaimoku, MD^*, Kana Maruyama, MD^*, Chizuru Saito, MD^*, Takamasa Katagiri, PhD^*, Kohei Hosokawa, MD, PhD^*, Ken Ishiyama, MD, Ph.D., Hirohito Yamazaki, MD, PhD^* and Shinji Nakao, MD, PhD

Cellular Transplantation Biology, Kanazawa University Graduate School of Medical Science, Kanazawa, Japan