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

This study is the first to demonstrate long-term safety and efficacy of hepatocyte-directed gene therapy in a large animal model. We conclude that hepatocyte-directed ex-vivo gene therapy is a rational choice for further exploration as an alternative therapeutic approach to whole organ transplantation for metabolic liver disease, including HT1.
PMID: 30477316 [PubMed - as supplied by publisher]

Source: Cell Transplantation - November 26, 2018 Category: Cytology Authors: Hickey RD, Nicolas CT, Allen K, Mao S, Elgilani F, Glorioso J, Amiot B, VanLith C, Guthman R, Du Z, Chen H, Harding CO, Kaiser RA, Nyberg SL, Lillegard JB Tags: Cell Transplant Source Type: research