Proteinopathies result from the proteasome not acting efficiently enough to eliminate harmful proteins and prevent the formation of the pathogenic aggregates. As described herein, inhibition of proteasome-associated deubiquitinase Usp14 results in increased proteasome efficiency. The present invention therefore provides novel compositions and methods for inhibition of Usp14, enhancement of proteasome activity and treatment of proteinopathies.
蛋白病症是由于
蛋白酶体不能有效地消除有害蛋白质并防止致病聚集体的形成。如本文所述,抑制
蛋白酶体相关的去泛素化酶 Usp14 可提高
蛋白酶体的效率。因此,本发明提供了抑制 Usp14、增强
蛋白酶体活性和治疗蛋白质病的新型组合物和方法。