机译:氟亚氨基糖醇对引起GM1 sub>-神经节苷脂病和Morquio B病的GLB1等位基因的基因产物具有有效的药理伴侣作用
Department of Pediatrics, Medical University of Graz, Graz, Austria;
Glycogroup, Department of Organic Chemistry, Graz University of Technology, Graz, Austria;
Glycogroup, Department of Organic Chemistry, Graz University of Technology, Graz, Austria;
Department of Pediatrics, Medical University of Graz, Graz, Austria;
Department of Pediatrics, Medical University of Graz, Graz, Austria;
机译:氟亚氨基糖醇对GLB基因产物起有效的药理伴侣作用。导致GM1神经节病和Morquio B病的等位基因。
机译:人类GLB1敲除脑类器官:测试AAV9介导的GLB1基因治疗减少GM1神经节病中GM1神经节苷脂贮藏的模型系统
机译:在GM1神经节病和Morquio B患者中发现的14个GLB1突变等位基因的表达和特征
机译:人类GLB1敲除脑类器官:测试AAV9介导的GLB1基因治疗减少GM1神经节病中GM1神经节苷脂贮藏的模型系统
机译:GM1神经节病和Morquio B疾病:基因改变和临床发现的最新进展