نتایج جستجو برای: mngie

تعداد نتایج: 83  

Journal: :The Journal of clinical investigation 2003
Yutaka Nishigaki Ramon Martí William C Copeland Michio Hirano

Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) is an autosomal recessive disorder caused by loss-of-function mutations in the gene encoding thymidine phosphorylase (TP). This deficiency of TP leads to increased circulating levels of thymidine (deoxythymidine, dThd) and deoxyuridine (dUrd) and has been associated with multiple deletions and depletion of mitochondrial DNA (mtDNA). ...

Journal: :Neurology 2013
Bridget E Bax Murray D Bain Mauro Scarpelli Massimiliano Filosto Paola Tonin Nicholas Moran

Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) is a rare autosomal recessive metabolic disorder caused by a deficiency of thymidine phosphorylase (TP, EC2.4.2.4) due to mutations in the nuclear gene TYMP. TP deficiency leads to plasma and tissue accumulations of thymidine and deoxyuridine which generate imbalances within themitochondrial nucleotide pools, ultimately leading to mi...

Journal: :Journal of controlled release : official journal of the Controlled Release Society 2009
Caroline De Vocht An Ranquin Ronnie Willaert Jo A Van Ginderachter Tamara Vanhaecke Vera Rogiers Wim Versées Patrick Van Gelder Jan Steyaert

The lack of a crucial metabolic enzyme can lead to accumulating substrate concentrations in the bloodstream and severe human enzyme deficiency diseases. Mitochondrial Neurogastrointestinal Encephalomyopathy (MNGIE) is such a fatal genetic disorder, caused by a thymidine phosphorylase deficiency. Enzyme replacement therapy is a strategy where the deficient enzyme is administered intravenously in...

Journal: :Biochimica et Biophysica Acta (BBA) - General Subjects 2012

Journal: :Annals of Clinical and Translational Neurology 2020

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