Curative ex vivo liver-directed gene therapy in a pig model of hereditary tyrosinemia type 1

We tested the hypothesis that ex vivo hepatocyte gene therapy can correct the metabolic disorder in fumarylacetoacetate hydrolase–deficient (Fah(−/−)) pigs, a large animal model of hereditary tyrosinemia type 1 (HT1). Recipient Fah(−/−)pigs underwent partial liver resection and hepatocyte isolation...

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Veröffentlicht in:Sci Transl Med
Hauptverfasser: Hickey, Raymond D., Mao, Shennen A., Glorioso, Jaime, Elgilani, Faysal, Amiot, Bruce, Chen, Harvey, Rinaldo, Piero, Marler, Ronald, Jiang, Huailei, DeGrado, Timothy R., Suksanpaisan, Lukkana, O’Connor, Michael K., Freeman, Brittany L., Ibrahim, Samar H., Peng, Kah Whye, Harding, Cary O., Ho, Chak-Sum, Grompe, Markus, Ikeda, Yasuhiro, Lillegard, Joseph B., Russell, Stephen J., Nyberg, Scott L.
Format: Artigo
Sprache:Inglês
Veröffentlicht: 2016
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Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5477771/
https://ncbi.nlm.nih.gov/pubmed/27464750
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/scitranslmed.aaf3838
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