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

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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Bibliographic Details
Main Authors: 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: Article
Language:English
Published: SAGE Publishing 2019-01-01
Series:Cell Transplantation
Online Access:https://doi.org/10.1177/0963689718814188

Internet

https://doi.org/10.1177/0963689718814188

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