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@ARTICLE{Peifer:127281,
author = {M. Peifer and F. Hertwig$^*$ and F. Roels$^*$ and D.
Dreidax$^*$ and M. Gartlgruber$^*$ and R. Menon and A.
Krämer and J. L. Roncaioli and F. Sand and J. M. Heuckmann
and F. Ikram and R. Schmidt and S. Ackermann and A. Engesser
and Y. Kahlert and W. Vogel and J. Altmüller and P.
Nürnberg and J. Thierry-Mieg and D. Thierry-Mieg and A.
Mariappan and S. Heynck and E. Mariotti and K.-O.
Henrich$^*$ and C. Gloeckner and G. Bosco and I. Leuschner
and M. R. Schweiger and L. Savelyeva$^*$ and S. C. Watkins
and C. Shao and E. Bell and T. Höfer and V. Achter and U.
Lang and J. Theissen and R. Volland and M. Saadati$^*$ and
A. Eggert and B. de Wilde and F. Berthold and Z. Peng and C.
Zhao and L. Shi and M. Ortmann and R. Büttner and S. Perner
and B. Hero and A. Schramm and J. Schulte$^*$ and C.
Herrmann$^*$ and R. J. O'Sullivan and F. Westermann$^*$ and
R. K. Thomas and M. Fischer$^*$},
title = {{T}elomerase activation by genomic rearrangements in
high-risk neuroblastoma.},
journal = {Nature},
volume = {526},
number = {7575},
issn = {1476-4687},
address = {London [u.a.]},
publisher = {Nature Publ. Group},
reportid = {DKFZ-2017-03306},
pages = {700 - 704},
year = {2015},
abstract = {Neuroblastoma is a malignant paediatric tumour of the
sympathetic nervous system. Roughly half of these tumours
regress spontaneously or are cured by limited therapy. By
contrast, high-risk neuroblastomas have an unfavourable
clinical course despite intensive multimodal treatment, and
their molecular basis has remained largely elusive. Here we
have performed whole-genome sequencing of 56 neuroblastomas
(high-risk, n = 39; low-risk, n = 17) and discovered
recurrent genomic rearrangements affecting a chromosomal
region at 5p15.33 proximal of the telomerase reverse
transcriptase gene (TERT). These rearrangements occurred
only in high-risk neuroblastomas (12/39, $31\%)$ in a
mutually exclusive fashion with MYCN amplifications and ATRX
mutations, which are known genetic events in this tumour
type. In an extended case series (n = 217), TERT
rearrangements defined a subgroup of high-risk tumours with
particularly poor outcome. Despite a large structural
diversity of these rearrangements, they all induced massive
transcriptional upregulation of TERT. In the remaining
high-risk tumours, TERT expression was also elevated in
MYCN-amplified tumours, whereas alternative lengthening of
telomeres was present in neuroblastomas without TERT or MYCN
alterations, suggesting that telomere lengthening represents
a central mechanism defining this subtype. The 5p15.33
rearrangements juxtapose the TERT coding sequence to strong
enhancer elements, resulting in massive chromatin
remodelling and DNA methylation of the affected region.
Supporting a functional role of TERT, neuroblastoma cell
lines bearing rearrangements or amplified MYCN exhibited
both upregulated TERT expression and enzymatic telomerase
activity. In summary, our findings show that remodelling of
the genomic context abrogates transcriptional silencing of
TERT in high-risk neuroblastoma and places telomerase
activation in the centre of transformation in a large
fraction of these tumours.},
keywords = {Chromatin (NLM Chemicals) / MYCN protein, human (NLM
Chemicals) / N-Myc Proto-Oncogene Protein (NLM Chemicals) /
Nuclear Proteins (NLM Chemicals) / Oncogene Proteins (NLM
Chemicals) / RNA, Messenger (NLM Chemicals) / TERT protein,
human (NLM Chemicals) / Telomerase (NLM Chemicals) / DNA
Helicases (NLM Chemicals) / ATRX protein, human (NLM
Chemicals)},
cin = {B087 / B086 / C060 / B080 / L101 / L201},
ddc = {070},
cid = {I:(DE-He78)B087-20160331 / I:(DE-He78)B086-20160331 /
I:(DE-He78)C060-20160331 / I:(DE-He78)B080-20160331 /
I:(DE-He78)L101-20160331 / I:(DE-He78)L201-20160331},
pnm = {312 - Functional and structural genomics (POF3-312)},
pid = {G:(DE-HGF)POF3-312},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:26466568},
pmc = {pmc:PMC4881306},
doi = {10.1038/nature14980},
url = {https://inrepo02.dkfz.de/record/127281},
}