Gene Modified Blood Stem Cell Transplants Correct Neurologic Enzyme Deficits

Cells can cross the blood brain barrier and in graft and white cells become engrafted in the brain where they're known as microglia. This is exactly the principle of transplantation in this type of disease and it's known as cross-correction. The engrafted donor cells make and secrete enzyme that enzyme is taken up by neighboring cells that remain genetically enzyme deficient but are able to metabolize within their cytoplasm accumulated substrate. And so gene modified brain engrafted microglia make the enzyme that the patient needs and forever.