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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Détails bibliographiques
Publié dans:Sci Transl Med
Auteurs principaux: 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
Langue:Inglês
Publié: 2016
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Article
Accès en ligne: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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