#$%^&*AU2019200903A120191024.pdf#####ABSTRACT A method for treating a disease caused by protein retention in the endoplasmic reticulum (ER) with a sarcoplasmic/endoplasmic reticulum calcium ATPase pump inhibitor encapsulated in a polymer nanoparticle. The polymer nanoparticle is surfacemodified such that it is targeted to the ER. The inhibitor reduces protein retention in the 5 ER and the encapsulation lowers side effects of the inhibitor, e.g., cytotoxicity, as compared to administering the inhibitor without encapsulation. Also disclosed is a pharmaceutical composition that can be used for carrying out the method. Further provided is a transgenic mouse carrying in its genome a heterologous nucleic acid that encodes an H338Y mutant gp9lphx protein. The transgenic mouse can serve as a model 10 for human chronic granulomatous disease.115 ~1.5-* 0 -1.0. W-0 0). T0 00 0I - E 0 0 >；%0 0.0. "01 * 21.X106 0- 1X106 0 M5X 0 00 Fig. IC
