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@ARTICLE{Mulder:868119,
author = {Mulder, Inge A. and Rubio-Beltran, Eloísa and Ibrahimi,
Khatera and Dzyubachyk, Oleh and Khmelinskii, Artem and
Hoehn, Mathias and Terwindt, Gisela M. and Wermer, Marieke
J. H. and MaassenVanDenBrink, Antoinette and van den
Maagdenberg, Arn M. J. M},
title = {{I}ncreased {M}ortality and {V}ascular {P}henotype in a
{K}nock-{I}n {M}ouse {M}odel of {R}etinal {V}asculopathy
{W}ith {C}erebral {L}eukoencephalopathy and {S}ystemic
{M}anifestations},
journal = {Stroke},
volume = {51},
number = {1},
issn = {1524-4628},
address = {Philadelphia, Pa.},
publisher = {Lippincott Williams $\&$ Wilkins},
reportid = {FZJ-2019-06706},
pages = {300-307},
year = {2020},
abstract = {Background and Purpose—Retinal vasculopathy with cerebral
leukoencephalopathy and systemic manifestations (RVCL-S) is
an autosomal dominant small vessel disease caused by
C-terminal frameshift mutations in the TREX1 gene that
encodes the major mammalian 3′ to 5′ DNA exonuclease.
RVCL-S is characterized by vasculopathy, especially in
densely vascularized organs, progressive retinopathy,
cerebral microvascular disease, white matter lesions, and
migraine, but the underlying mechanisms are
unknown.Methods—Homozygous transgenic RVCL-S knock-in mice
expressing a truncated Trex1 (three prime repair exonuclease
1) protein (similar to what is seen in patients) and
wild-type littermates, of various age groups, were subjected
to (1) a survival analysis, (2) in vivo postocclusive
reactive hyperemia and ex vivo Mulvany myograph studies to
characterize the microvascular and macrovascular reactivity,
and (3) experimental stroke after transient middle cerebral
artery occlusion with neurological deficit
assessment.Results—The mutant mice show increased
mortality starting at midlife (P=0.03 with hazard ratio,
3.14 $[95\%$ CI, 1.05–9.39]). The mutants also show a
vascular phenotype as evidenced by attenuated postocclusive
reactive hyperemia responses (across all age groups; F[1,
65]=5.7, P=0.02) and lower acetylcholine-induced relaxations
in aortae (in 20- to 24-month-old mice; RVCL-S knock-in:
Emax: $37±8\%$ versus WT: Emax: $65±6\%,$ P=0.01). A
vascular phenotype is also suggested by the increased
infarct volume seen in 12- to 14-month-old mutant mice at 24
hours after infarct onset (RVCL-S knock-in: 75.4±2.7 mm3
versus WT: 52.9±5.6 mm3, P=0.01).Conclusions—Homozygous
RVCL-S knock-in mice show increased mortality, signs of
abnormal vascular function, and increased sensitivity to
experimental stroke and can be instrumental to investigate
the pathology seen in patients with RVCL-S.},
cin = {INM-3},
ddc = {610},
cid = {I:(DE-Juel1)INM-3-20090406},
pnm = {572 - (Dys-)function and Plasticity (POF3-572)},
pid = {G:(DE-HGF)POF3-572},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:31805844},
UT = {WOS:000504225600056},
doi = {10.1161/STROKEAHA.119.025176},
url = {https://juser.fz-juelich.de/record/868119},
}