T he lysosome, which was described by De Duve et al. in 1955, is an organelle with many important functions, including innate immunity and cell division; however, lysosomes may sometimes be the target of dysfunction, which leads to disruption of these important functions. To date, more than 50 lysosomal storage disorders (LSDs) have been identified as a result of the wide range of dysfunctions. Although there is currently no cure for these disorders, research has led to a rapid increase in medical treatments and therapies (Parkinson-Lawrence et al., 2010).
ETIOLOGYLSDs are a group of more than 50 diverse metabolic disorders caused by genetic deficits (i.e., missing enzymes) in lysosomal acid hydrolase, receptor, activator, membrane, or transporter proteins. Specific LSDs may be determined depending on the type of deficits in the lysosomal enzymes, cofactors, and transporters (Kadali et al., 2014), and they are typically categorized by the type of substrate stored. These issues cause progressive accumulation of substrates, which in turn results in the deterioration of cell and tissue functioning (Wang et al., 2011). They are single-gene disorders (Beck, 2018) and typically inherited in an autosomal recessive manner, with the exception of Fabry, Hunter, and Danon diseases, which are linked to the X chromosome (Wang et al., 2011).