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Extracellular vesicles from human liver stem cells restore argininosuccinate synthase deficiency
BACKGROUND: Argininosuccinate synthase (ASS)1 is a urea cycle enzyme that catalyzes the conversion of citrulline and aspartate to argininosuccinate. Mutations in the ASS1 gene cause citrullinemia type I, a rare autosomal recessive disorder characterized by neonatal hyperammonemia, elevated citrullin...
Autores principales: | Herrera Sanchez, Maria Beatriz, Previdi, Sara, Bruno, Stefania, Fonsato, Valentina, Deregibus, Maria Chiara, Kholia, Sharad, Petrillo, Sara, Tolosano, Emanuela, Critelli, Rossana, Spada, Marco, Romagnoli, Renato, Salizzoni, Mauro, Tetta, Ciro, Camussi, Giovanni |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2017
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5531104/ https://www.ncbi.nlm.nih.gov/pubmed/28750687 http://dx.doi.org/10.1186/s13287-017-0628-9 |
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