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    • Preclinical Efficacy and Safety Evaluation of Hematopoietic Stem Cell Gene Therapy in a Mouse Model of MNGIE 

      Yadak, Rana; Cabrera-Pérez, Raquel; Torres-Torronteras, Javier; Bugiani, Marianna; Haeck, Joost C; Huston, Marshall W; Bogaerts, Elly; Goffart, Steffi; Jacobs, Edwin H; Stok, Merel; Leonardelli, Lorena; Biasco, Luca; Verdijk, Robert M; Bernsen, Monique R;, Ruijter, George; Martí, Ramon; Wagemaker, Gerard; van Til, Niek P; de Coo, Irenaeus F M (Elsevier BV, 2018)
      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 ...