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Thymidine phosphorylase replacement has been achieved by allogeneic hematopoietic stem cell transplantation, a procedure hampered by high mortality. Based on high thymidine phosphorylase expression in the liver, a 25\u2010year\u2010old severely affected patient underwent liver transplantation. Serum levels of toxic nucleosides rapidly normalized. At 400 days of follow\u2010up, the patient's clinical conditions are stable. We propose liver transplantation as a new therapy for MNGIE. 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