Autologous Gene and Cell Therapy Provides Safe and Long-Term Curative Therapy in A Large Pig Model of Hereditary Tyrosinemia Type 1

Orthotopic liver transplantation remains the only curative therapy for inborn errors of metabolism. Given the tremendous success for primary immunodeficiencies using ex-vivo gene therapy with lentiviral vectors, there is great interest in developing similar curative therapies for metabolic liver dis...

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發表在:Cell Transplant
Main Authors: Hickey, Raymond D., Nicolas, Clara T., Allen, Kari, Mao, Shennen, Elgilani, Faysal, Glorioso, Jaime, Amiot, Bruce, VanLith, Caitlin, Guthman, Rebekah, Du, Zeji, Chen, Harvey, Harding, Cary O., Kaiser, Robert A., Nyberg, Scott L., Lillegard, Joseph B.
格式: Artigo
語言:Inglês
出版: SAGE Publications 2018
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Original Articles
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC6322137/
https://ncbi.nlm.nih.gov/pubmed/30477316
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1177/0963689718814188
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