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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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Detalles Bibliográficos
Autores principales:	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.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	SAGE Publications 2018
Materias:	Original Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6322137/ https://www.ncbi.nlm.nih.gov/pubmed/30477316 http://dx.doi.org/10.1177/0963689718814188

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