THERAPY FOR THE SPHINGOLIPIDOSES

Sphingolipidoses are human metabolic storage disorders characterized b y the accumulation of harmful quantities of glycosphingolipids and pho sphosphingolipids. These lipids have in common a hydrophobic portion o f their structure called ceramide. In glycosphingolipids, various olig osaccharides are linked to ceramide through glycosidic bonds. An examp le is glucocerebroside, composed of ceramide and 1 molecule of glucose . Large quantities of glucocerebroside accumulate in tissues in patien ts with Gaucher disease. Higher oligosaccharide homologues contain add itional neutral and acidic oligosaccharides. Among these are gangliosi des that have 1 or more molecules of N-acetylneuraminic acid. A gangli oside called G(M2) accumulates in Tay-Sachs disease. Sphingomyelin is a phosphosphingolipid that accumulates in patients with Niemann-Pick d isease.