Gene Therapy Using a Liver-targeted AAV Vector Restores Nucleoside and Nucleotide Homeostasis in a Murine Model of MNGIE

Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) is an autosomal recessive disorder caused by mutations in TYMP, enconding thymidine phosphorylase (TP). TP deficiency results in systemic accumulation of thymidine and deoxyuridine, which interferes with mitochondrial DNA (mtDNA) replicat...

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Detalhes bibliográficos
Main Authors: Torres-Torronteras, Javier, Viscomi, Carlo, Cabrera-Pérez, Raquel, Cámara, Yolanda, Di Meo, Ivano, Barquinero, Jordi, Auricchio, Alberto, Pizzorno, Giuseppe, Hirano, Michio, Zeviani, Massimo, Martí, Ramon
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2014
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4015233/
https://ncbi.nlm.nih.gov/pubmed/24448160
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2014.6
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