ABSTRACT
In this chapter the unique and crucially important organelle known as the lysosome is discussed. The function and composition of the lysosome are detailed, along with its somewhat serendipitous discovery by Nobel Prize winners Christian de Duve, Albert Claude, and George Palade. Lysosome-related organelles and lysosomal storage disorders such as Tay Sachs and others are discussed. Also addressed in the chapter are the newly developing treatments for lysosomal storage disorders, including Gaucher disease and Niemann–Pick disease. Although CRISPR technology has not yet led to approved treatments for lysosomal storage disorders, another form of therapy known as enzyme replacement therapy has some success in providing purified enzyme to cells to replace the missing or dysfunctional enzyme. The chapter again highlights how basic understanding of lysosomes and their function has led to inventive treatments that help patients.
