Lysosomal storage diseases

Abstract

Lysosomes are subcellular organelles responsible for the physiologic turnover of cell constituents. Lysosomal storage diseases describe a heterogeneous group of dozens of rare inherited disorders characterized by the accumulation of undigested or partially digested macromolecules, which ultimately results in cellular dysfunction and clinical abnormalities. Several studies have explored different methods of treatment, but only are promising and safe even though they are very expensive.