Reduction of Cholesterol in Humanized Mice Using CRISPR/CAS9

Humanized Mouse. We conduct our experiments on a mouse model that has its hepatocytes (liver cells) repopulated with human cells. This allows us to test our gene editing in human cells, in-vivo. Goal. Our goal is to lower cholesterol by knocking out 4 genes with crispr/Cas9 as a potential therapy. Results. Huh7 cells were grown to 70% confluence and transduced with purified recombinant adenovirus. A multiplicity of infection (moi) of 1000 and 1500 viral particles per cell was used. The heavy upper bands correspond to the unedited, wild-type gene while the lower bands correspond to the successful excision of the gene of interest. This project was completed with contributions from Francis Pankowicz, Xavier Legras, Mercedes Barzi, Nikal Juricic, Collin Johnson, Eleanor Davis, Corinne Sonnet, Colin King, Beatrice Bissig-Choisat, Elizabeth A. Olmstead-Davis, William R. Lagor and Karl-Dimiter Bissig from the Baylor College of Medicine, Houston.