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 surface-modified such that it is targeted to the ER. The inhibitor reduces protein retention in the 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 gp91phox protein. The transgenic mouse can serve as a model for human chronic granulomatous disease.一種用囊封於聚合物奈米粒子中之肌質/內質網鈣離子ATP酶泵抑制劑治療由內質網(ER)中之蛋白質駐留所引起之疾病的方法。該聚合物奈米粒子經表面修飾以使得其靶向該ER。該抑制劑減少該ER中之蛋白質駐留，且相較於投予未經囊封之該抑制劑，囊封會減少該抑制劑之副作用，例如細胞毒性。亦揭露一種可用於實施該方法之醫藥組成物。進一步提供一種在基因組中攜帶編碼H338Y突變型gp91phox蛋白質之異源核酸的基因轉殖小鼠。該基因轉殖小鼠可充當人類慢性肉芽腫病之模型。
