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Overview of HSCT approaches
Graphical summary of HSCT using either allogeneic donor cells, or genetically modified patient cells to secrete a supraphysiological level of the defective enzyme. HSCT mediates therapeutic effect in the central nervous system by an HSC subpopulation crossing the BBB, engrafting the CNS, and generating genetically modified microglia, which provides a source of therapeutic enzyme to cross-correct neighboring enzyme-deficient brain cells.
