Hematopoietic gene therapy restores thymidine phosphorylase activity in a cell culture and a murine model of MNGIE

Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) is an autosomal recessive disorder caused by mutations in the TYMP gene, which encodes thymidine phosphorylase (TP). TP dysfunction results in systemic thymidine (dThd) and deoxyuridine (dUrd) overload, which selectively impair mitochondr...

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Publicado en:Gene Ther
Main Authors: Torres-Torronteras, J, Gómez, A, Eixarch, H, Palenzuela, L, Pizzorno, G, Hirano, M, Andreu, AL, Barquinero, J, Martí, R
Formato: Artigo
Idioma:Inglês
Publicado: 2011
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Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC7568345/
https://ncbi.nlm.nih.gov/pubmed/21451581
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2011.24
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