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@ARTICLE{Mearelli:282568,
author = {Mearelli, Marika and Hirschberg, Insa and Weissleder,
Christin and Giachino, Carmela and Pérez, María José and
Dubroux, Malvina and Provenzano, Francesca and Rizzuti,
Mafalda and Ottoboni, Linda and Sheth, Udit and Gendron,
Tania F and Corti, Stefania and Deleidi, Michela},
title = {{C}9orf72 {R}epeat {E}xpansion {I}nduces {M}etabolic
{D}ysfunction in {H}uman i{PSC}-{D}erived {M}icroglia and
{M}odulates {G}lial-{N}euronal {C}rosstalk.},
journal = {Glia},
volume = {74},
number = {1},
issn = {0894-1491},
address = {Bognor Regis [u.a.]},
publisher = {Wiley-Liss},
reportid = {DZNE-2025-01331},
pages = {e70080},
year = {2026},
abstract = {The C9orf72 hexanucleotide repeat expansion mutation is the
most common genetic cause of amyotrophic lateral sclerosis
(ALS) and frontotemporal dementia, but its cell
type-specific effects on energy metabolism and immune
pathways remain poorly understood. Using induced pluripotent
stem cell (iPSC)-derived motor neurons, astrocytes, and
microglia from C9orf72 patients and their isogenic controls,
we investigated metabolic changes at the single-cell level
under basal and inflammatory conditions. Our results showed
that microglia are particularly susceptible to metabolic
disturbances. While C9orf72 motor neurons exhibited impaired
mitochondrial respiration and reduced ATP production,
C9orf72 microglia presented pronounced increases in
glycolytic activity and oxidative stress, accompanied by the
upregulation of the expression of key metabolic enzymes.
These metabolic changes in microglia were exacerbated by
inflammatory stimuli. To investigate how these changes
affect the broader cellular environment, we developed a
human iPSC-derived triculture system comprising motor
neurons, astrocytes, and microglia. This model revealed
increased metabolic activity in all cell types and
highlighted that microglia-driven metabolic reprogramming in
astrocytes contributes to the vulnerability of motor neurons
under inflammatory conditions. Our findings highlight the
central role of microglia in driving metabolic dysregulation
and intercellular crosstalk in ALS pathogenesis and suggest
that targeting metabolic pathways in immune cells may
provide new therapeutic avenues.},
keywords = {Humans / C9orf72 Protein: genetics / C9orf72 Protein:
metabolism / Microglia: metabolism / Induced Pluripotent
Stem Cells: metabolism / Motor Neurons: metabolism /
Amyotrophic Lateral Sclerosis: genetics / Amyotrophic
Lateral Sclerosis: metabolism / Astrocytes: metabolism / DNA
Repeat Expansion: genetics / Cells, Cultured / Cell
Communication: physiology / Oxidative Stress / C9orf72
(Other) / amyotrophic lateral sclerosis/frontotemporal
dementia (Other) / glial‐neuronal communication (Other) /
immune system (Other) / induced pluripotent stem cells
(Other) / microglia (Other) / C9orf72 Protein (NLM
Chemicals) / C9orf72 protein, human (NLM Chemicals)},
cin = {AG Deleidi},
ddc = {610},
cid = {I:(DE-2719)1210011},
pnm = {352 - Disease Mechanisms (POF4-352)},
pid = {G:(DE-HGF)POF4-352},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:40888599},
pmc = {pmc:PMC12667002},
doi = {10.1002/glia.70080},
url = {https://pub.dzne.de/record/282568},
}