Article
Therapeutic strategies to ameliorate lysosomal storage disorders--a focus on Gaucher disease.
Cellular and molecular life sciences : CMLS - 1 May 2006
Sawkar A R, D'Haeze W, Kelly J W
Abstract excerpt
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...
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