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Emerging Strategies for the Treatment of Hereditary Metabolic Storage Disorders

17 May 2006

Abstract excerpt

Metabolic storage disorders are caused by mutations in genes that result in insufficient activity of enzymes required for the catabolism of substances that arise from the turnover of senescent cells in the body. Among the most prevalent of these conditions are Gaucher disease and Fabry disease, which are caused by reduced activity of the housekeeping enzymes glucocerebrosidase and alpha-galactosidase A,...

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PMID: 16706651
DOI: 10.1089/rej.2006.9.237

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