Hepatotoxicity and Toxicology of Lentiviral Vector Administration in Healthy and Liver-Injury Mouse Models.

General safety and toxicology assessments supporting lentiviral vector-based therapeutic development are sparse. We have previously demonstrated the efficacy of a lentiviral vector expressing fumarylacetoacetate hydrolase (LV-FAH) to cure animal models of hereditary tyrosinemia type 1. Therefore, we performed a complete preclinical toxicological evaluation of LV-FAH, in a large cohort ( = 20/group) of wildtype mice and included matched groups of N-nitrosodiethylamine/carbon tetrachloride (DEN/CCl)-induced liver injury mice to assess specific toxicity in fibrotic liver tissue.