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©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
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 mice | Mice | BM-MSCs | IV | Improve degenerative loss of Purkinje neurons | [194] |
BALB/c npcnih (NPC) mice | Mice | BM-MSCs | Cerebellum injection | Promote neuronal networks with functional synaptic transmission | [195] |
BALB/c npcnih (NPC) mice | Mice | BM-MSCs | Cerebellum injection | Upregulate fusion ability of Purkinje neurons and donor-derived BM-MSCs | [196] |
BALB/c npcnih (NPC) mice | Mice | BM-MSCs | Cerebrum injection | Modulate endogenous NPC NSCs, stimulate NSC proliferation and neuronal differentiation | [199] |
BALB/c npcnih (NPC) mice | Mice | BM-MSCs | Cerebellum injection | Inhibit activation of astrocytes and microglia, reduce inflammation | [197] |
BALB/c npcnih (NPC) mice | Mice | BM-MSCs | Cerebellum injection | Release bioactive neurotrophic factors, modulate sphingolipid metabolism of endogenous NPC Purkinje neurons | [198] |
BALB/c npcnih (NPC) mice | Mice | ADSCs | Cerebellum injection | Rescue Purkinje neurons, restore motor coordination, and alleviate inflammatory responses | [201] |
BALB/c npcnih (NPC) mice | Human | hUC-MSCs | HIPP injection | Stimulate endogenous neurogenesis, diminish intracellular cholesterol accumulation, and safeguard motor functionality | [200] |
BALB/c npcnih (NPC) mice | Human | hUC-MSCs | IN | Reduce cholesterol levels, decrease loss of Purkinje cells in the cerebellum, delay motor dysfunction | [202] |
Npc1KO N2a cells | Human | MenSCs-CM | Coculture | Increase survival rate, significantly alleviate inflammatory responses and apoptosis | [203] |
- Citation: Cui CX, Shao XN, Li YY, Qiao L, Lin JT, Guan LH. Therapeutic potential of mesenchymal stem cells in neurodegenerative diseases. World J Stem Cells 2025; 17(8): 107717
- URL: https://www.wjgnet.com/1948-0210/full/v17/i8/107717.htm
- DOI: https://dx.doi.org/10.4252/wjsc.v17.i8.107717