English: Figure 3. Current lysosomal storage disorder therapeutic strategies. Gene therapy and gene editing approaches aim to introduce a functional gene or to correct the defective gene or transcript inside cells. Hematopoietic stem cell transplantation (HSCT) repopulates specific tissues and allows local release of functional lysosomal hydrolases by healthy donor cells. Substrate reduction therapy (SRT) inhibits substrate synthase at the early Golgi compartment. Enzyme replacement therapies (ERTs) deliver functional enzymes to lysosomes via the endocytosis pathway. Chaperones can stabilize the mutant enzyme and partially restore catalytic activity in the endoplasmic reticulum (ER) and overall in lysosomes. Lastly, proteostasis modifiers stabilize transcription factors within the nucleus, improve lysosomal enzyme expression and translation in the ER, reduce lysosomal membrane permeability, and improve lysosomal function.