Non-human mammal comprising a modified SERCA2 gene and methods, cells, genes, and vectors thereof

摘要

The present invention relates to a non-human mammal in which all cells contain genetic modifications in a Ca2+ handling Serca ATPase gene, more particularly the Serca2 ATPase. Defective Ca2+ handling is induced in live animals by introducing genetic elements which direct the timing and specificity of the gene inactivation to the organ or tissue of interest; e.g. cardiac muscle cells. The primary application is to provide a standardized and reproducible animal model for heart failure or other human diseases, in which the defective Ca2+ handling function by the Serca gene can be manipulated in live animals. This is the only existing animal to date with such genetic modifications for this class of Ca2+ handling proteins.