Experimental Variables that Affect Human Hepatocyte AAV Transduction in Liver Chimeric Mice.

ABSTRACT: Adeno-associated virus (AAV) vector serotypes vary in their ability to transduce hepatocytes from different species. Chimeric mouse models harboring human hepatocytes have shown translational promise for liver-directed gene therapies. However, many variables that influence human hepatocyte transduction and transgene expression in such models remain poorly defined. Here, we aimed to test whether three experimental conditions influence AAV transgene expression in immunodeficient, fumaryl-acetoactetate-hydrolase-deficient (Fah ^-/-) chimeric mice repopulated with primary human hepatocytes. We examined the effects of the murine liver injury cycle, human donor variability, and vector doses on hepatocyte transduction with various AAV serotypes expressing a green fluorescent prot

SUBMITTER: Zou C 

PROVIDER: S-EPMC7326722 | biostudies-literature | 2020 Sep

REPOSITORIES: biostudies-literature