Hematopoietic stem cell therapy has been tried for several years now. It has become much better. It still has its benefits and its drawbacks. The benefit is that it replaces the enzyme that is missing. You have to do one transplant, and then if it works then you’re set because your body now has a new tissue, a foreign tissue that has that enzyme which will degrade that substrate. But it comes with the cost of immunosuppression, it comes with a high mortality rate.
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