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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書誌詳細
出版年:Sci Transl Med
主要な著者: 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.
フォーマット: Artigo
言語:Inglês
出版事項: 2016
主題:
Article
オンライン･アクセス: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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