Preclinical Efficacy and Safety Evaluation of Hematopoietic Stem Cell Gene Therapy in a Mouse Model of MNGIE - Yadak, Rana (Department of Hematology, Erasmus University Medical Center, Rotterdam, the Netherlands) ; Cabrera-Pérez, Raquel (Hospital Universitari Vall d'Hebron) ; Torres-Torronteras, Javier (Hospital Universitari Vall d'Hebron) ; Bugiani, Marianna (Department of Pathology, VU University Medical Center, Amsterdam, the Netherlands) ; Haeck, Joost C. (Department of Radiology & Nuclear Medicine, Erasmus University Medical Center, Rotterdam, the Netherlands) ; Huston, Marshall W. (Department of Neurology, Erasmus University Medical Center, Rotterdam, the Netherlands) ; Bogaerts, Elly (Department of Clinical Genetics, Erasmus University Medical Center, Rotterdam, the Netherlands) ; Goffart, Steffi (Department of Biology, University of Eastern Finland, Joensuu, Finland) ; Jacobs, Edwin H. (Department of Clinical Genetics, Erasmus University Medical Center, Rotterdam, the Netherlands) ; Stok, Merel (Department of Pediatrics, Erasmus University Medical Center, Rotterdam, the Netherlands) ; Leonardelli, Lorena (San Raffaele Telethon Institute for Gene Therapy (Milà, Itàlia)) ; Biasco, Luca (University College of London (UCL), Great Ormond Street Institute of Child Health (ICH), London, UK) ; Verdijk, Robert M. (Department of Pathology, Erasmus University Medical Center, Rotterdam, the Netherlands) ; Bernsen, Monique R. (Department of Radiology & Nuclear Medicine, Erasmus University Medical Center, Rotterdam, the Netherlands) ; Ruijter, George (Department of Clinical Genetics, Erasmus University Medical Center, Rotterdam, the Netherlands) ; Martí, Ramon A. (Hospital Universitari Vall d'Hebron) ; Wagemaker, Gerard (Raisa Gorbacheva Memorial Research Institute for Pediatric Oncology and Hematology, Saint Petersburg, Russia) ; van Til, Niek P. (Laboratory of Translational Immunology, University Medical Center Utrecht, Utrecht, the Netherlands) ; de Coo, Irenaeus F. M. (Department of Neurology, Erasmus University Medical Center, Rotterdam, the Netherlands) ; Universitat Autònoma de Barcelona