Inhibition of substrate synthesis: a pharmacological approach for glycosphingolipid storage disease therapy
The neuronopathic lysosomal storage diseases are not currently amenable to therapy due to the difficulties of delivering functional enzyme/protein to the brain. Over two decades ago, an alternative strategy was suggested by Radin based on small-molecule enzyme inhibitors for treating a subset of these disorders, the glycosphingolipidoses. Recently, the efficacy of this approach has been demonstrated in animal disease models and in the clinic. This chapter discusses these studies. It begins with a brief background on glycosphingolipids (GSLs) and GSL storage diseases, as it provides insights into the potentials and the limitations of drug-based strategies for treating this family of predominantly neurodegenerative disorders.
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