造血幹細胞
Hematopoietic stem cell (HSC) transplantation is another treatment for lysosomal storage diseases. HSCs are derived from bone-marrow. These cells have the ability to mature into the many cell types that comprise blood, including red blood cells, platelets, and white blood cells. Patients suffering from enzyme deficiencies often undergo HSC transplantations in which HSCs from a healthy donor are injected. This treatment introduces HSCs that regularly produce the deficient enzyme since they have normal metabolic function. This treatment is often used to treat the central nervous system of patients with some lysosomal storage diseases.