Emptying the stores: lysosomal diseases and therapeutic strategies

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...

Bibliografske podrobnosti
Glavni avtor:	Platt, F
Format:	Journal article
Izdano:	Springer Nature 2017

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