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Cross-disorder and disease-specific pathways in dementia revealed by single-cell genomics
Journal article   Peer reviewed

Cross-disorder and disease-specific pathways in dementia revealed by single-cell genomics

Jessica E. Rexach, Yuyan Cheng, Lawrence Chen, Damon Polioudakis, Li-Chun Lin, Vivianne Mitri, Andrew Elkins, Xia Han, Mai Yamakawa, Anna Yin, …
Cell, Vol.187(20), pp.5753-5774.e28
10/2024
DOI: 10.1016/j.cell.2024.08.019
PMCID: PMC12017262
PMID: 39265576
url
https://pmc.ncbi.nlm.nih.gov/articles/PMC12017262/pdf/nihms-2055870.pdfView
Open Access

Abstract

The development of successful therapeutics for dementias requires an understanding of their shared and distinct molecular features in the human brain. We performed single-nuclear RNA-seq and ATAC-seq in Alzheimer’s disease (AD), frontotemporal dementia (FTD), and progressive supranuclear palsy (PSP), analyzing 41 participants and ∼1 million cells (RNA + ATAC) from three brain regions varying in vulnerability and pathological burden. We identify 32 shared, disease-associated cell types and 14 that are disease specific. Disease-specific cell states represent glial-immune mechanisms and selective neuronal vulnerability impacting layer 5 intratelencephalic neurons in AD, layer 2/3 intratelencephalic neurons in FTD, and layer 5/6 near-projection neurons in PSP. We identify disease-associated gene regulatory networks and cells impacted by causal genetic risk, which differ by disorder. These data illustrate the heterogeneous spectrum of glial and neuronal compositional and gene expression alterations in different dementias and identify therapeutic targets by revealing shared and disease-specific cell states. [Display omitted] •Perform comparative genomic analysis of AD, bvFTD, and PSP at the single-cell level•Pinpoint markers and candidate drivers of selective neuronal vulnerability in dementia•Identify disorder-specific microglia, astrocyte, and oligodendrocyte glial-immune states•Causal genetic risk impacts disorder-specific gene regulatory networks and cells Functional genomic analyses of multiple brain regions across three major neurodegenerative disorders involving tau pathology, AD, bvFTD, and PSP, at the single-cell level enables the identification of markers of neuronal vulnerability, glial states that vary across disease, and disorder-specific cellular differences in the expression and regulation of known-risk genes.
drug discovery functional genomics KCNH7 MAFG multi-omics NFE2L1 NLGN1 OPCML PDE1C tauopathy

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