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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...
| Autores principales: | , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
2015
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| Materias: | |
| 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 |