Boston University School of Medicine
Pharmacology & Experimental Therapeutics
Dr. Julia TCW is an assistant professor in the Department of Pharmacology and Experimental Therapeutics and a director of the Laboratory of Human Induced Pluripotent Stem Cell Therapeutics at Boston University School of Medicine. Dr. TCW received Ph.D. and A.M. in Molecular and Cellular Biology from Harvard University with research studies in induced pluripotent stem cell (iPSC) reprogramming in the Department of Stem Cell and Regenerative Biology. She then was a Druckenmiller Fellow awarded from New York Stem Cell Foundation and became a faculty in the Department of Neuroscience, Genetics and Genomic Sciences and Ronald M. Loeb Center for Alzheimer’s Disease at Icahn School of Medicine at Mount Sinai, New York. Dr. TCW’s research program utilized iPSC technology and forward genetic approach to identify molecular and cellular dysfunctions in human brains by Alzheimer’s disease (AD) genetic risk. Her ongoing work includes identifying cell type specific defects by Apolipoprotein E4 (APOE4) risk and developing in vitro and in vivo human brain model system by engineering human genome. She is also collaborating with the industry to find a drug target for AD.

Moderator of 1 Session

Presenter of 1 Presentation

APOE Ε4 DRIVEN HUMAN SPECIFIC CHOLESTEROL TRAFFICKING DYSREGULATION IN GLIA

Session Type
SYMPOSIUM
Date
Thu, 17.03.2022
Session Time
02:45 PM - 04:45 PM
Room
ONSITE: 112
Lecture Time
03:45 PM - 04:00 PM

Abstract

Aims

The association of Apolipoprotein E (APOE) ɛ4 with Alzheimer's disease risk is well-established however, the impact of APOE ɛ4 on human brain cell function remains unclear. We hypothesized that human APOE ε4/ε4 genotype contributes to disease risk through cell autonomous and non-cell autonomous mechanisms in a human specific manner.

Methods

Global transcriptomic analyses were performed on astrocytes, microglia, mixed cortical cultures (neurons and astrocytes) and brain microvascular endothelial cells derived from human induced pluripotent stem cells (iPSCs), post-mortem brains and microglia and astrocytes from APOE targeted replacement (TR) mice. Lipid pathway dysregulation identified through GSEA, WGCNA and cell type deconvolution was validated in vitro using Gas Chromatography-Mass Spectrometry (GC-MS) to measure intracellular cholesterol and filipin staining with immunocytochemistry and western blotting to determine the subcellular compartment that accumulates cholesterol in iPSC-based models, and further confirmed by immunohistochemical analysis in human APOE4 post-mortem brain.

Results

Global transcriptomic analyses reveal that APOE 44 drives lipid metabolic dysregulation in astrocytes and microglia from human iPSCs and cell type-deconvolved post-mortem AD brain but not in primary murine APOE 44 astrocytes and microglia. APOE ε4 leads to elevated de novo cholesterol synthesis despite the intracellular accumulation of free cholesterol coupled with decreased efflux due to lysosomal cholesterol sequestration in iPSC-derived astrocytes and in hippocampal astrocytes from APOE ε4 human brain.

Conclusions

Human APOE ε4 causes human glia-specific, region-specific, cell autonomous lipid dysregulation that may increase AD risk.

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