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iPSC-derived neural precursor cells engineering GBA1 recovers acid β-glucosidase deficiency and diminishes α-synuclein and neuropathology.
Mol Ther Methods Clin Dev. 2023 Mar 15;29:185-201. doi: 10.1016/j.omtm.2023.03.007. eCollection 2023 Jun 8.
Mol Ther Methods Clin Dev. 2023.
PMID: 37063480
Free PMC article.
Analysis of the Biomarkers for Neurodegenerative Diseases in Aged Progranulin Deficient Mice.
Zhao X, Hasan S, Liou B, Lin Y, Sun Y, Liu C.
Zhao X, et al.
Int J Mol Sci. 2022 Jan 6;23(2):629. doi: 10.3390/ijms23020629.
Int J Mol Sci. 2022.
PMID: 35054815
Free PMC article.
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Systemic enzyme delivery by blood-brain barrier-penetrating SapC-DOPS nanovesicles for treatment of neuronopathic Gaucher disease.
Sun Y, Liou B, Chu Z, Fannin V, Blackwood R, Peng Y, Grabowski GA, Davis HW, Qi X.
Sun Y, et al.
EBioMedicine. 2020 May;55:102735. doi: 10.1016/j.ebiom.2020.102735. Epub 2020 Apr 10.
EBioMedicine. 2020.
PMID: 32279952
Free PMC article.
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Progranulin associates with Rab2 and is involved in autophagosome-lysosome fusion in Gaucher disease.
Zhao X, Liberti R, Jian J, Fu W, Hettinghouse A, Sun Y, Liu CJ.
Zhao X, et al.
J Mol Med (Berl). 2021 Nov;99(11):1639-1654. doi: 10.1007/s00109-021-02127-6. Epub 2021 Aug 27.
J Mol Med (Berl). 2021.
PMID: 34453183
Free PMC article.
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Substrate Reduction Therapy Reverses Mitochondrial, mTOR, and Autophagy Alterations in a Cell Model of Gaucher Disease.
Peng Y, Liou B, Lin Y, Fannin V, Zhang W, Feldman RA, Setchell KDR, Grabowski GA, Sun Y.
Peng Y, et al.
Cells. 2021 Sep 2;10(9):2286. doi: 10.3390/cells10092286.
Cells. 2021.
PMID: 34571934
Free PMC article.
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Intravenous infusion of iPSC-derived neural precursor cells increases acid β-glucosidase function in the brain and lessens the neuronopathic phenotype in a mouse model of Gaucher disease.
Peng Y, Liou B, Inskeep V, Blackwood R, Mayhew CN, Grabowski GA, Sun Y.
Peng Y, et al.
Hum Mol Genet. 2019 Oct 15;28(20):3406-3421. doi: 10.1093/hmg/ddz184.
Hum Mol Genet. 2019.
PMID: 31373366
Free PMC article.
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