One of the most well-known applications of SRT is in the treatment of lysosomal storage diseases (LSDs). In these conditions, specific substrates accumulate due to deficient enzymatic activity in lysosomes. For example, in Gaucher disease, glucocerebroside accumulates due to a deficiency in the enzyme glucocerebrosidase. SRT, through the use of drugs like miglustat, reduces the synthesis of glucocerebroside, thereby alleviating the symptoms of the disease.
