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@ARTICLE{Kettwig:163450,
author = {Kettwig, Matthias and Ternka, Katharina and Wendland,
Kristin and Krüger, Dennis Manfred and Zampar, Silvia and
Schob, Charlotte and Franz, Jonas and Aich, Abhishek and
Winkler, Anne and Sakib, M Sadman and Kaurani, Lalit and
Epple, Robert and Werner, Hauke B and Hakroush, Samy and
Kitz, Julia and Prinz, Marco and Bartok, Eva and Hartmann,
Gunther and Schröder, Simone and Rehling, Peter and
Henneke, Marco and Boretius, Susann and Alia, A. and Wirths,
Oliver and Fischer, Andre and Stadelmann, Christine and
Nessler, Stefan and Gärtner, Jutta},
title = {{I}nterferon-driven brain phenotype in a mouse model of
{RN}ase{T}2 deficient leukoencephalopathy.},
journal = {Nature Communications},
volume = {12},
number = {1},
issn = {2041-1723},
address = {[London]},
publisher = {Nature Publishing Group UK},
reportid = {DZNE-2022-00210},
pages = {6530},
year = {2021},
note = {(CC BY)},
abstract = {Infantile-onset RNaseT2 deficient leukoencephalopathy is
characterised by cystic brain lesions, multifocal white
matter alterations, cerebral atrophy, and severe psychomotor
impairment. The phenotype is similar to congenital
cytomegalovirus brain infection and overlaps with type I
interferonopathies, suggesting a role for innate immunity in
its pathophysiology. To date, pathophysiological studies
have been hindered by the lack of mouse models
recapitulating the neuroinflammatory encephalopathy found in
patients. In this study, we generated Rnaset2-/- mice using
CRISPR/Cas9-mediated genome editing. Rnaset2-/- mice
demonstrate upregulation of interferon-stimulated genes and
concurrent IFNAR1-dependent neuroinflammation, with
infiltration of CD8+ effector memory T cells and
inflammatory monocytes into the grey and white matter.
Single nuclei RNA sequencing reveals homeostatic
dysfunctions in glial cells and neurons and provide
important insights into the mechanisms of
hippocampal-accentuated brain atrophy and cognitive
impairment. The Rnaset2-/- mice may allow the study of CNS
damage associated with RNaseT2 deficiency and may be used
for the investigation of potential therapies.},
keywords = {Animals / CD8-Positive T-Lymphocytes: metabolism /
Cognitive Dysfunction: genetics / Cognitive Dysfunction:
metabolism / Disease Models, Animal / Endoribonucleases:
genetics / Endoribonucleases: metabolism / Female / Flow
Cytometry / Genotype / Humans / Immunohistochemistry /
Leukoencephalopathies: genetics / Leukoencephalopathies:
metabolism / Leukoencephalopathies: pathology / Magnetic
Resonance Imaging / Male / Memory T Cells: metabolism / Mice
/ Mice, Knockout / Neuroglia: metabolism / Real-Time
Polymerase Chain Reaction / Endoribonucleases (NLM
Chemicals)},
cin = {Bioinformatics and Genome Dynamics Core / AG Fischer},
ddc = {500},
cid = {I:(DE-2719)1440016 / I:(DE-2719)1410002},
pnm = {352 - Disease Mechanisms (POF4-352)},
pid = {G:(DE-HGF)POF4-352},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:34764281},
pmc = {pmc:PMC8586222},
doi = {10.1038/s41467-021-26880-x},
url = {https://pub.dzne.de/record/163450},
}