Review
Copyright ©The Author(s) 2025.
World J Stem Cells. Aug 26, 2025; 17(8): 107717
Published online Aug 26, 2025. doi: 10.4252/wjsc.v17.i8.107717
Table 6 Summary of therapeutic efficacy and safety of mesenchymal stem cells and their derivatives in Niemann-Pick disease type C
Animal model
Source
Type
Administration method
Main outcomes
Ref.
NP-C GsbsGFP miceMiceBM-MSCsIVImprove degenerative loss of Purkinje neurons[194]
BALB/c npcnih (NPC) miceMiceBM-MSCsCerebellum injectionPromote neuronal networks with functional synaptic transmission[195]
BALB/c npcnih (NPC) miceMiceBM-MSCsCerebellum injectionUpregulate fusion ability of Purkinje neurons and donor-derived BM-MSCs[196]
BALB/c npcnih (NPC) miceMiceBM-MSCsCerebrum injectionModulate endogenous NPC NSCs, stimulate NSC proliferation and neuronal differentiation[199]
BALB/c npcnih (NPC) miceMiceBM-MSCsCerebellum injectionInhibit activation of astrocytes and microglia, reduce inflammation[197]
BALB/c npcnih (NPC) miceMiceBM-MSCsCerebellum injectionRelease bioactive neurotrophic factors, modulate sphingolipid metabolism of endogenous NPC Purkinje neurons[198]
BALB/c npcnih (NPC) miceMiceADSCsCerebellum injectionRescue Purkinje neurons, restore motor coordination, and alleviate inflammatory responses[201]
BALB/c npcnih (NPC) miceHumanhUC-MSCsHIPP injectionStimulate endogenous neurogenesis, diminish intracellular cholesterol accumulation, and safeguard motor functionality[200]
BALB/c npcnih (NPC) miceHumanhUC-MSCsINReduce cholesterol levels, decrease loss of Purkinje cells in the cerebellum, delay motor dysfunction[202]
Npc1KO N2a cellsHumanMenSCs-CMCocultureIncrease survival rate, significantly alleviate inflammatory responses and apoptosis[203]