Lysosomal Storage Disorders

Lysosomal storage disorders are a group of inherited metabolic diseases caused by defects in lysosomal function. These defects lead to the accumulation of undigested molecules within the lysosomes, causing cell damage and a variety of symptoms depending on the specific disorder. Examples include Gaucher disease, Fabry disease, and Tay-Sachs disease. Treatment options may include enzyme replacement therapy, substrate reduction therapy, and gene therapy.

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