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. 2022 Dec 22;18:1143–1155. doi: 10.2147/TCRM.S351300

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© 2022 Poswar et al.

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Therapeutic approaches for MPS VII. Enzyme replacement therapy leads to significant improvement in patient’s quality of life, with disease correction in many visceral tissues; however, it is costly, requires the support of health centers, and there is the possibility of immune reactions. Hematopoietic stem cell transplantation, on the other hand, is effective in the brain, though it must be performed early in life, it takes time for cells to fully engraft and be effective, a matching donor is necessary, and there is always a life-threatening risk of graft rejection or graft-versus-host disease. Finally, gene therapy should be a once-in-a-lifetime treatment (or a few times throughout the lifespan of the patient), able to target the central nervous system specifically and improve cognition significantly, although it can be a very invasive procedure with associated risks inherent to viral vectors.