Substrate reduction therapy for glycosphingolipid storage disorders.
Substrate reduction therapy is a novel approach to treating glycosphingolipid (GSL) lysosomal storage disorders. These diseases are caused by mutations in the genes coding for enzymes involved in GSL catabolism and are characterised by the accumulation of GSL substrates within the lysosomes of cells...
| Main Authors: | , |
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| Format: | Journal article |
| Language: | English |
| Published: |
2001
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