Lithium ameliorates Niemann-Pick C1 disease phenotypes by impeding STING/SREBP2 activation
Han S, Wang Q, Song Y, Pang M, Ren C, Wang J, Guan D, Xu W, Li F, Wang F, Zhou X, Fernández-Hernando C, Zhang H, Wu D, Ye Z. Lithium ameliorates Niemann-Pick C1 disease phenotypes by impeding STING/SREBP2 activation. IScience 2023, 26: 106613. PMID: 37128603, PMCID: PMC10148154, DOI: 10.1016/j.isci.2023.106613.Peer-Reviewed Original ResearchTherapeutic optionsSTING activationPotential therapeutic optionProgressive neurodegenerative phenotypeNiemann-Pick disease type CGenetic lysosomal disorderNP-C patientsCytosolic calcium concentrationDisease type CEffects of lithiumCerebellar inflammationLithium treatmentMouse modelLithium effectsLysosomal disordersNeurodegenerative phenotypeCalcium concentrationSREBP2 activationDisease phenotypeType CMiceSREBP2 pathwayActivationDeficient fibroblastsPhenotype