Expression and Retention of Thymidine Phosphorylase in Cultured Reticulocytes as a Novel Treatment for MNGIE

Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) is a rare autosomal metabolic disorder caused by thymidine phosphorylase (TP) deficiency. Successful therapeutic interventions for this disease rely on a means for efficient and long-lasting circulation of the TP enzyme. In this study we...

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Detalhes bibliográficos
Publicado no:Mol Ther Methods Clin Dev
Main Authors: Meinders, Marjolein, Shoemark, Debbie, Dobbe, Johannes G.G., Streekstra, Geert J., Frayne, Jan, Toye, Ashley M.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2020
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7191122/
https://ncbi.nlm.nih.gov/pubmed/32368563
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.03.029
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