Infantile neuronal ceroid lipofuscinosis (INCL) is often a fatal the neurodegenerative disorder brought on by a deficiency from the lysosomal enzyme palmitoyl protein thioesterase-1 (PPT1). Ppt1 knockout mice show hallmarks of INCL and mimic the human pathology: accumulation of lipofuscin, Smoothened degeneration of CNS neurons, and a shortened lifestyle span. Purified non-genetically modified human CNS stem cells, grown as neurospheres (hCNS-SCns), have been transplanted to the brains of immunodeficient Ppt1(-/-) mice the place they engrafted robustly, migrated extensively, and made ample ranges of PPT1 to alter host neuropathology. Grafted mice displayed decreased autofluorescent lipofuscin, sizeable neuroprotection of host hippocampal and cortical neurons, and delayed reduction of motor coordination. Early intervention with cellular transplants of hCNS-SCns to the brains of INCL individuals may well supply a constant and long-lasting supply of the missing PPT1 and present some therapeutic benefit through safety of endogenous neurons. These information supply the experimental basis for human clinical trials with these banked hCNS-SCns.