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SHORT POLYPEPTIDE MEDIATED CONFORMATIONAL CHANGES PROMOTING ENHANCEMENT OF ENZYMATIC ACTIVITY

机译:短肽介导的构象变化,促进酶活性的增强

摘要

The invention provides for an isolated and purified tagged dimeric protein as well as a process for producing and purifying the same and the use thereof. The present invention also relates to methods and pharmaceutical compositions for the treatment of lysosomal storage diseases. More specifically the invention provides methods and compositions for treating sulfatase deficiency. In particular, the invention relates to methods and compositions for treating sulfatase deficiency diseases caused all or in part by deficiencies in human N-acetylgalactosamine-6-sulfatase (GALNS) including mucopolysaccharidosis IVA (MPS IVA).
机译:本发明提供了分离的和纯化的带标签的二聚体蛋白及其产生和纯化的方法及其用途。本发明还涉及用于治疗溶酶体贮积病的方法和药物组合物。更具体地说,本发明提供了治疗硫酸酯酶缺乏症的方法和组合物。特别地,本发明涉及用于治疗全部或部分由人N-乙酰半乳糖胺-6-硫酸酶(GALNS)缺乏症(包括粘多糖贮积症IVA)(MPS IVA)引起的硫酸酯酶缺乏症的方法和组合物。

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