Mucopolysaccharidosis type II (MPS II), or Hunter syndrome, is an X-linked lysosomal storage disease caused by genetic deficiency of the enzyme iduronate-2-sulfatase (IDS), required for the step-wise degradation and recycling of complex glycosaminoglycans (GAGs). Loss of IDS activity results in abnormal accumulation of GAGs (i.e., heparan and dermatan sulfates) in multiple tissues and organs, resulting in progressive cellular and multi-organ dysfunction.<br/>Currently, the protein drug is highly expensive for enzyme replacement therapy (ERT) of MPS II. In order to cost down and have a better life quality of patients, it is necessary to discover a new approach by using small molecules for treating this GAG metabolic disease. However, because of the structural complication of GAGs, convenient evaluation platforms for small molecule screening toward MPSII remain to be explored.<br/>Herein, we would like to establish a series of analytical platforms including enzyme-based, cell-based assays for small molecules evaluation. Through our effort, several analytic parameters have been examined to establish these accessible systems. With these assays in hand, a small molecule library screening will be performed soon.

Establish cell-based assays for small molecules evaluation toward mucopolysaccharidosis type II

2019 Fall National Meeting

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