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@ARTICLE{Bremer:304113,
author = {J. Bremer and J. Nagel and J. Zschüntzsch and K. K. Zajt
and T. Palaz and T. Blank and A. Ikis and L. A. Fischer and
A. S. M. Sensmeyer and L. Wiechers and J. J. Reichelt and K.
Hofmann$^*$ and M. J. Wolf and C. Leuchtenberger$^*$ and P.
Tripathi and C. Einer and H. Zischka and U. Rothermel$^*$
and A.-L. Eck$^*$ and R. Reimann and V. Kana and E. Rushing
and A. Aguzzi and M. Prinz and D. Liebetanz and F. Odoardi
and C.-C. Kuo and J. Weis and F. Kraft and J. Schmidt and M.
Heikenwälder$^*$},
title = {{M}utual reinforcement of lymphotoxin-driven myositis and
impaired autophagy in murine muscle.},
journal = {Brain},
volume = {nn},
issn = {0006-8950},
address = {Oxford},
publisher = {Oxford Univ. Press},
reportid = {DKFZ-2025-01776},
pages = {nn},
year = {2025},
note = {#LA:D440# / epub},
abstract = {Inclusion body myositis (IBM) is a progressive muscle
disorder characterized by inflammation and degeneration with
altered proteostasis. To better understand the
interrelationship between these two features, we aimed at
establishing a novel preclinical mouse model. First, we used
quantitative PCR to determine expression of pro-inflammatory
chemo- and cytokines including lymphotoxin (LT)-signaling
pathway components in human skeletal muscle tissue diagnosed
with myositis. Based on these results we generated a mouse
model that we analyzed at the histological, ultrastructural,
transcriptional, biochemical, and behavioral level. Lastly,
we subjected this model to anti-inflammatory treatments.
After confirming and extending previous data on activation
of lymphotoxin (LT)-signaling in human myositis, we
generated distinct transgenic mouse lines co-expressing LTα
and -β in skeletal muscle fibers. Transgenic mice displayed
chronic myositis accompanied by dysregulated proteostasis,
including an altered autophagolysosomal pathway that
initially shows signs of activation and later exhaustion and
decreased flux. To enhance the latter, we genetically
impaired autophagy in skeletal muscle cells. Autophagy
impairment alone induced a pro-inflammatory transcriptional
state, but no obvious cellular inflammation. However, the
combination of LT-driven myositis with autophagy impairment
induced the full spectrum of characteristic molecular and
pathological features of IBM in skeletal muscle, including
protein inclusions with typical ultrastructural morphology
and mild mitochondrial pathology. Our attempts to treat the
pathology by subjecting these mice to corticosteroids or
anti-Thy1.2 antibodies mirrored recent treatment failures in
humans, i.e., none of these treatments resulted in
significant clinical improvement of motor performance or the
transcriptional profile of muscle pathology. In summary,
these data provide evidence that inflammation and autophagy
disruption play a synergistic role in the development of
IBM-like muscular pathology. Furthermore, once established,
IBM-like pathology in these mice, as in human IBM patients
cannot be reverted or prevented from progression by
conventional means of immunosuppression. We expect that this
novel mouse model will help to identify future treatment
modalities for IBM.},
keywords = {NF-κB signaling (Other) / autophagy (Other) / inclusion
body myositis (Other) / lymphotoxin (Other) / lymphotoxin
signaling (Other) / myositis (Other)},
cin = {D440},
ddc = {610},
cid = {I:(DE-He78)D440-20160331},
pnm = {314 - Immunologie und Krebs (POF4-314)},
pid = {G:(DE-HGF)POF4-314},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:40853947},
doi = {10.1093/brain/awaf260},
url = {https://inrepo02.dkfz.de/record/304113},
}