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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發表在:Sci Transl Med
Main Authors: 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.
格式: Artigo
語言:Inglês
出版: 2016
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在線閱讀: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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