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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...
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| Udgivet i: | Gene Ther |
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| Main Authors: | , , , , , |
| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
2015
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| Fag: | |
| Online adgang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4746739/ https://ncbi.nlm.nih.gov/pubmed/26609667 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2015.107 |
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