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  1. Preclinical Efficacy and Safety Evaluation of Hematopoietic Stem Cell Gene Therapy in a...

    © 2018 The Authors 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)

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