Safety and Efficacy of Erythrocyte Encapsulated Thymidine Phosphorylase in Mitochondrial Neurogastrointestinal Encephalomyopathy

Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) is an ultra-rare autosomal recessive disorder of nucleoside metabolism that is caused by mutations in the nuclear thymidine phosphorylase gene (TYMP) gene, encoding for the enzyme thymidine phosphorylase. There are currently no approved t...

詳細記述

保存先:
書誌詳細
出版年:J Clin Med
主要な著者: Levene, Michelle, Bain, Murray D., Moran, Nicholas F., Nirmalananthan, Niranjanan, Poulton, Joanna, Scarpelli, Mauro, Filosto, Massimiliano, Mandel, Hanna, MacKinnon, Andrew D., Fairbanks, Lynette, Pacitti, Dario, Bax, Bridget E
フォーマット: Artigo
言語:Inglês
出版事項: MDPI 2019
主題:
Article
オンライン･アクセス:https://ncbi.nlm.nih.gov/pmc/articles/PMC6517976/
https://ncbi.nlm.nih.gov/pubmed/30959750
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/jcm8040457
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