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@ARTICLE{Jensen:140836,
author = {Jensen, Lars R and Garrett, Lillian and Hölter, Sabine M
and Rathkolb, Birgit and Rácz, Ildikó and Adler, Thure and
Prehn, Cornelia and Hans, Wolfgang and Rozman, Jan and
Becker, Lore and Aguilar-Pimentel, Juan Antonio and Puk,
Oliver and Moreth, Kristin and Dopatka, Monika and Walther,
Diego J and von Bohlen Und Halbach, Viola and Rath, Matthias
and Delatycki, Martin and Bert, Bettina and Fink, Heidrun
and Blümlein, Katharina and Ralser, Markus and Van Dijck,
Anke and Kooy, Frank and Stark, Zornitza and Müller, Sabine
and Scherthan, Harry and Gecz, Jozef and Wurst, Wolfgang and
Wolf, Eckhard and Zimmer, Andreas and Klingenspor, Martin
and Graw, Jochen and Klopstock, Thomas and Busch, Dirk and
Adamski, Jerzy and Fuchs, Helmut and Gailus-Durner, Valérie
and de Angelis, Martin Hrabě and von Bohlen Und Halbach,
Oliver and Ropers, Hans-Hilger and Kuss, Andreas W},
title = {{A} mouse model for intellectual disability caused by
mutations in the {X}-linked 2'‑{O}‑methyltransferase
{F}tsj1 gene.},
journal = {Biochimica et biophysica acta / Molecular basis of disease},
volume = {1865},
number = {9},
issn = {0925-4439},
address = {Amsterdam},
publisher = {Elsevier},
reportid = {DZNE-2020-07158},
pages = {2083-2093},
year = {2019},
abstract = {Mutations in the X chromosomal tRNA
2'‑O‑methyltransferase FTSJ1 cause intellectual
disability (ID). Although the gene is ubiquitously expressed
affected individuals present no consistent clinical features
beyond ID. In order to study the pathological mechanism
involved in the aetiology of FTSJ1 deficiency-related
cognitive impairment, we generated and characterized an
Ftsj1 deficient mouse line based on the gene trapped stem
cell line RRD143. Apart from an impaired learning capacity
these mice presented with several statistically
significantly altered features related to behaviour, pain
sensing, bone and energy metabolism, the immune and the
hormone system as well as gene expression. These findings
show that Ftsj1 deficiency in mammals is not phenotypically
restricted to the brain but affects various organ systems.
Re-examination of ID patients with FTSJ1 mutations from two
previously reported families showed that several features
observed in the mouse model were recapitulated in some of
the patients. Though the clinical spectrum related to Ftsj1
deficiency in mouse and man is variable, we suggest that an
increased pain threshold may be more common in patients with
FTSJ1 deficiency. Our findings demonstrate novel roles for
Ftsj1 in maintaining proper cellular and tissue functions in
a mammalian organism.},
keywords = {Animals / Behavior, Animal / Cognition Disorders: etiology
/ Cognition Disorders: pathology / Disease Models, Animal /
Family / Female / Intellectual Disability: etiology /
Intellectual Disability: pathology / Male / Mental
Retardation, X-Linked: genetics / Methyltransferases:
genetics / Methyltransferases: metabolism /
Methyltransferases: physiology / Mice / Mice, Inbred C57BL /
Mice, Knockout / Mutation / Nociceptive Pain: etiology /
Nociceptive Pain: pathology / Nuclear Proteins: genetics /
Nuclear Proteins: metabolism / tRNA Methyltransferases:
genetics / tRNA Methyltransferases: metabolism / tRNA
Methyltransferases: physiology},
cin = {AG Wurst / Clinical Dementia Research München ; AG Levin},
ddc = {610},
cid = {I:(DE-2719)1140001 / I:(DE-2719)1111016},
pnm = {342 - Disease Mechanisms and Model Systems (POF3-342) / 344
- Clinical and Health Care Research (POF3-344)},
pid = {G:(DE-HGF)POF3-342 / G:(DE-HGF)POF3-344},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:30557699},
doi = {10.1016/j.bbadis.2018.12.011},
url = {https://pub.dzne.de/record/140836},
}