Emptying the stores: lysosomal diseases and therapeutic strategies
Lysosomal storage disorders (LSDs) — designated as 'orphan' diseases — are inborn errors of metabolism caused by defects in genes that encode proteins involved in various aspects of lysosomal homeostasis. For many years, LSDs were viewed as unattractive targets for the development of thera...
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| Formaat: | Journal article |
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Springer Nature
2017
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