Therapeutic strategies to ameliorate lysosomal storage disorders – a focus on Gaucher disease

  title={Therapeutic strategies to ameliorate lysosomal storage disorders – a focus on Gaucher disease},
  author={Anu R. Sawkar and Wim D’Haeze and Jeffery W Kelly},
  journal={Cellular and Molecular Life Sciences CMLS},
The lysosomal storage disorders encompass more than 40 distinct diseases, most of which are caused by the deficient activity of a lysosomal hydrolase leading to the progressive, intralysosomal accumulation of substrates such as sphingolipids, mucopolysaccharides, and oligosaccharides. Here, we primarily focus on Gaucher disease, one of the most prevalent lysosomal storage disorders, which is caused by an impaired activity of glucocerebrosidase, resulting in the accumulation of the… CONTINUE READING


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