Enzyme therapy is not enough

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Presentation transcript:

Enzyme therapy is not enough Prof Gregory A Grabowski, MD  The Lancet  Volume 358, (December 2001) DOI: 10.1016/S0140-6736(01)07042-8 Copyright © 2001 Elsevier Ltd Terms and Conditions

Figure Schematic diagram of glucocerebroside synthesis and degradative pathways The O-series, A-series, and B-series of higher glucosphingolipids comprise over 300 compounds. Newly synthesised glucocerebroside products incorporate into cellular membranes and extracellular structures. In the viscera, these cellular components are internalised and delivered to the lysosomes for degradation. Lac=lactosylceramide; cer=ceramide; GalNAcT=N-acetyl-galactosaminyl-transferase; glc=glucose. The Lancet 2001 358, DOI: (10.1016/S0140-6736(01)07042-8) Copyright © 2001 Elsevier Ltd Terms and Conditions

The Lancet 2001 358, DOI: (10.1016/S0140-6736(01)07042-8) Copyright © 2001 Elsevier Ltd Terms and Conditions