Lysosomes and Lysosomal Storage Disorders

The lysosome is the main site for catabolic recycling and it also has an important role in cellular homeostasis and other cellular processes. Lysosomal storage diseases (LSDs) are caused by defects in genes encoding components of the lysosomal system. The resultant disruption of a catabolic pathway leads to retention of partially degraded molecules or digestion products in lysosomes and consequently to lysosomal hypertrophy and a cascade of secondary pathophysiology. Progress in our understanding of the biochemical, genetic and cellular basis of LSDs has led to the development of several potential therapeutic approaches, some of which have reached the clinic. 
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