Article
High Dose Versus Low Dose Syngeneic Hepatocyte Transplantation in Pex1-G844D NMRI Mouse Model is Safe but Does Not Achieve Long Term Engraftment.
Cells - 30 Dec 2020
Demaret Tanguy, Evraerts Jonathan, Ravau Joachim, Roumain Martin, Muccioli Giulio G, Najimi Mustapha, Sokal Etienne M
Abstract excerpt
Genetic alterations in PEX genes lead to peroxisome biogenesis disorder. In humans, they are associated with Zellweger spectrum disorders (ZSD). No validated treatment has been shown to modify the dismal natural history of ZSD. Liver transplantation (LT) improved clinical and biochemical outcomes in mild ZSD patients. Hepatocyte transplantation (HT), developed to overcome LT limitations, was performed in a mild...
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