Large-scale network analysis of the cerebrospinal fluid proteome identifies molecular signatures of frontotemporal lobar degeneration
(2025) In Nature Aging- Abstract
The pathophysiological mechanisms driving disease progression of frontotemporal lobar degeneration (FTLD) and corresponding biomarkers are not fully understood. Here we leveraged aptamer-based proteomics (>4,000 proteins) to identify dysregulated communities of co-expressed cerebrospinal fluid proteins in 116 adults carrying autosomal dominant FTLD mutations (C9orf72, GRN and MAPT) compared with 39 non-carrier controls. Network analysis identified 31 protein co-expression modules. Proteomic signatures of genetic FTLD clinical severity included increased abundance of RNA splicing (particularly in C9orf72 and GRN) and extracellular matrix (particularly in MAPT) modules, as well as decreased abundance of synaptic/neuronal and autophagy... (More)
The pathophysiological mechanisms driving disease progression of frontotemporal lobar degeneration (FTLD) and corresponding biomarkers are not fully understood. Here we leveraged aptamer-based proteomics (>4,000 proteins) to identify dysregulated communities of co-expressed cerebrospinal fluid proteins in 116 adults carrying autosomal dominant FTLD mutations (C9orf72, GRN and MAPT) compared with 39 non-carrier controls. Network analysis identified 31 protein co-expression modules. Proteomic signatures of genetic FTLD clinical severity included increased abundance of RNA splicing (particularly in C9orf72 and GRN) and extracellular matrix (particularly in MAPT) modules, as well as decreased abundance of synaptic/neuronal and autophagy modules. The generalizability of genetic FTLD proteomic signatures was tested and confirmed in independent cohorts of (1) sporadic progressive supranuclear palsy-Richardson syndrome and (2) frontotemporal dementia spectrum clinical syndromes. Network-based proteomics hold promise for identifying replicable molecular pathways in adults living with FTLD. ‘Hub’ proteins driving co-expression of affected modules warrant further attention as candidate biomarkers and therapeutic targets.
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- author
- author collaboration
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- publishing date
- 2025
- type
- Contribution to journal
- publication status
- epub
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- in
- Nature Aging
- publisher
- Springer
- external identifiers
-
- pmid:40380000
- scopus:105005430750
- DOI
- 10.1038/s43587-025-00878-2
- language
- English
- LU publication?
- yes
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- Publisher Copyright: © The Author(s), under exclusive licence to Springer Nature America, Inc. 2025.
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- 803ce6b3-8702-428e-8780-84e4df3cc6ed
- date added to LUP
- 2025-05-26 06:48:21
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- 2025-07-07 10:20:46
@article{803ce6b3-8702-428e-8780-84e4df3cc6ed, abstract = {{<p>The pathophysiological mechanisms driving disease progression of frontotemporal lobar degeneration (FTLD) and corresponding biomarkers are not fully understood. Here we leveraged aptamer-based proteomics (>4,000 proteins) to identify dysregulated communities of co-expressed cerebrospinal fluid proteins in 116 adults carrying autosomal dominant FTLD mutations (C9orf72, GRN and MAPT) compared with 39 non-carrier controls. Network analysis identified 31 protein co-expression modules. Proteomic signatures of genetic FTLD clinical severity included increased abundance of RNA splicing (particularly in C9orf72 and GRN) and extracellular matrix (particularly in MAPT) modules, as well as decreased abundance of synaptic/neuronal and autophagy modules. The generalizability of genetic FTLD proteomic signatures was tested and confirmed in independent cohorts of (1) sporadic progressive supranuclear palsy-Richardson syndrome and (2) frontotemporal dementia spectrum clinical syndromes. Network-based proteomics hold promise for identifying replicable molecular pathways in adults living with FTLD. ‘Hub’ proteins driving co-expression of affected modules warrant further attention as candidate biomarkers and therapeutic targets.</p>}}, author = {{Saloner, Rowan and Staffaroni, Adam M. and Dammer, Eric B. and Johnson, Erik C.B. and Paolillo, Emily W. and Wise, Amy and Heuer, Hilary W. and Forsberg, Leah K. and Lario-Lago, Argentina and Webb, Julia D. and Vogel, Jacob W. and Santillo, Alexander F. and Hansson, Oskar and Kramer, Joel H. and Miller, Bruce L. and Li, Jingyao and Loureiro, Joseph and Sivasankaran, Rajeev and Worringer, Kathleen A. and Seyfried, Nicholas T. and Yokoyama, Jennifer S. and Spina, Salvatore and Grinberg, Lea T. and Seeley, William W. and VandeVrede, Lawren and Ljubenkov, Peter A. and Bayram, Ece and Bozoki, Andrea and Brushaber, Danielle and Considine, Ciaran M. and Day, Gregory S. and Dickerson, Bradford C. and Domoto-Reilly, Kimiko and Faber, Kelley and Galasko, Douglas R. and Gendron, Tania and Geschwind, Daniel H. and Ghoshal, Nupur and Graff-Radford, Neill and Hales, Chadwick M. and Honig, Lawrence S. and Hsiung, Ging Yuek R. and Huey, Edward D. and Kornak, John and Kremers, Walter and Lapid, Maria I. and Lee, Suzee E. and Litvan, Irene and McMillan, Corey T. and Mendez, Mario F. and Miyagawa, Toji and Pantelyat, Alexander and Pascual, Belen and Masdeu, Joseph and Paulson, Henry L and Petrucelli, Leonard and Pressman, Peter and Rademakers, Rosa and Marisa Ramos, Eliana and Rascovsky, Katya and Roberson, Erik D. and Savica, Rodolfo and Snyder, Allison and Campbell Sullivan, Anna and Tartaglia, M. Carmela and Vandebergh, Marijne and Boeve, Brad F. and Rosen, Howie J. and Rojas, Julio C. and Boxer, Adam L. and Casaletto, Kaitlin B.}}, language = {{eng}}, publisher = {{Springer}}, series = {{Nature Aging}}, title = {{Large-scale network analysis of the cerebrospinal fluid proteome identifies molecular signatures of frontotemporal lobar degeneration}}, url = {{http://dx.doi.org/10.1038/s43587-025-00878-2}}, doi = {{10.1038/s43587-025-00878-2}}, year = {{2025}}, }