Helper-dependent adenoviral vectors for liver-directed gene therapy of primary hyperoxaluria type 1

Primary hyperoxaluria type 1 (PH1) is an inborn error of liver metabolism due to deficiency of the peroxisomal enzyme alanine:glyoxylate aminotransferase (AGT) which catalyzes conversion of glyoxylate into glycine. AGT deficiency results in overproduction of oxalate which ultimately leads to end-sta...

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Detalles Bibliográficos
Autores principales: Castello, Raffaele, Borzone, Roberta, D’Aria, Stefania, Annunziata, Patrizia, Piccolo, Pasquale, Brunetti-Pierri, Nicola
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2015
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4746739/
https://www.ncbi.nlm.nih.gov/pubmed/26609667
http://dx.doi.org/10.1038/gt.2015.107

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4746739/
https://www.ncbi.nlm.nih.gov/pubmed/26609667
http://dx.doi.org/10.1038/gt.2015.107

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