Stem cell transplantation in neurodegenerative diseases including Alzheimer’s disease has been reported. In this study, we hypothesized that human umbilical cord blood-derived multipotent stem cells (hUCB-MSCs) have the ability to differentiate into functional neurons in the brain. To test this hypothesis, we verifi ed this assumption in a mouse model of Niemann-Pick type C (NPC1-/-) disease using hUCB-MSCs. hUCB-MSCs were transplanted into the hippocampus of asymptomatic mice with NPC1-/- disease. The transplantation resulted in the recovery of motor function and dramatically prolonged the survival of the NPC1-/- mice. Interestingly, many of the transplanted hUCBMSCs showed microtubule associated protein 2-positive cells with electrophysiological function in the brains of the NPC1- /- mice. To examine if the hUCB-MSCs affect endogenous cell survival in the brain, we performed western blotting using mice brains. These results showed that the hUCB-MSCs can activate the Phosphoinositide 3-kinase-Akt-glycogen synthase kinase 3 beta ser9 and JAK2-Stat3 pathways for neuronal survival and regeneration in the brain by secreting functional factors. We concluded that hUCB-MSCs could contribute to extending the life of the NPC1-/-mice with the recovery of motor function by the differentiation into functional active neurons and cell survival signal cascade. Therefore, it is suggested that hUCBMSCs may be applicable in a broad range of neurodegenerative diseases.
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