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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Auteurs principaux: Raymond D. Hickey, Clara T. Nicolas, Kari Allen, Shennen Mao, Faysal Elgilani, Jaime Glorioso, Bruce Amiot, Caitlin VanLith, Rebekah Guthman, Zeji Du, Harvey Chen, Cary O. Harding, Robert A. Kaiser, Scott L. Nyberg, Joseph B. Lillegard
Format: Artigo
Langue:Inglês
Publié: SAGE Publishing 2019-01-01
Collection:Cell Transplantation
Accès en ligne:https://doi.org/10.1177/0963689718814188
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