Genetically Modified Mouse Model for Human Hepatocyte Xenotransplantation

摘要

One or more embodiments of the present invention include an immunodeficient mouse genetically modified to include a gene encoding a PiZ variant (Glu342Lys) of human alpha-1 antitrypsin (AAT), wherein the mouse is human alpha-1 antitrypsin (Z-AAT) and has a reduced number of mouse hepatocytes compared to immunodeficient mice of the same type that do not express Z-AAT. An immunodeficient mouse genetically modified to include a gene encoding a PiZ variant of human AAT may be a genetically modified NSG, NRG or NOG mouse. The immunodeficient mouse may further include heterologous hepatocytes, such as human hepatocytes. Predictive treatment of human liver disease can be evaluated in mice provided according to aspects of the present disclosure.