1.
|
14 p, 2.3 MB |
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. Institut de Recerca) ;
Torres-Torronteras, Javier (Hospital Universitari Vall d'Hebron. Institut de Recerca) ;
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. Institut de Recerca) ;
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
Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) is an autosomal recessive disorder caused by thymidine phosphorylase (TP) deficiency resulting in systemic accumulation of thymidine (d-Thd) and deoxyuridine (d-Urd) and characterized by early-onset neurological and gastrointestinal symptoms. [...]
2018 - 10.1016/j.omtm.2018.01.001
Molecular Therapy. Methods & Clinical Development, Vol. 8 (january 2018) , p. 152-165
|
|