智鼠故事 
Gaa KO
复苏/繁育服务
代谢及心血管模型
* 使用本品系发表的文献需注明：Gaa KO mice (Catalog C001702) were purchased from Cyagen.
交付类型
周龄
性别
基因型
数量
只
品系描述
溶酶体酸性α-葡萄糖苷酶（GAA）基因编码的溶酶体酸性α-葡萄糖苷酶是介导溶酶体内糖原水解降解的关键酶。Gaa KO 小鼠是基因敲除（KO）模型，利用基因编辑技术敲除了小鼠体内 Gaa 基因（人类 GAA 基因的同源基因）的蛋白编码序列。该模型可用于糖原贮积病 II 型（庞贝病）的致病机制研究以及相关治疗手段的研发。
相关疾病：
Glycogen Storage Disease Ii
Glycogen Storage Disease Due to Acid Maltase Deficiency, Late-Onset
Glycogen Storage Disease
Glucosidase Acid-1,4-Alpha Deficiency
Glycogen Storage Disease Due to Acid Maltase Deficiency, Infantile Onset
Glycogen Storage Disease Iv
Glycogen Storage Disease Ia
Glycogen Storage Disease Due to Glucose-6-Phosphatase Deficiency
Creatine Phosphokinase, Elevated Serum
Isolated Elevated Serum Creatine Phosphokinase Levels
Myopathy
应用领域
溶酶体糖原代谢研究
糖原贮积病 II 型（庞贝病）致病机制研究及治疗药物评估
