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@ARTICLE{Grschel:143513,
author = {S. Gröschel$^*$ and D. Hübschmann$^*$ and F. Raimondi and
P. Horak$^*$ and G. Warsow$^*$ and M. Fröhlich$^*$ and B.
Klink and L. Gieldon$^*$ and B. Hutter$^*$ and K.
Kleinheinz$^*$ and D. Bonekamp$^*$ and O. Marschal and P.
Chudasama$^*$ and J. Mika$^*$ and M. Groth and S. Uhrig$^*$
and S. Krämer$^*$ and C. Heining and C. Heilig$^*$ and D.
Richter$^*$ and E. Reisinger$^*$ and K. Pfütze$^*$ and R.
Eils$^*$ and S. Wolf$^*$ and C. von Kalle$^*$ and C.
Brandts$^*$ and C. Scholl$^*$ and W. Weichert$^*$ and S.
Richter and S. Bauer$^*$ and R. Penzel$^*$ and E.
Schröck$^*$ and A. Stenzinger$^*$ and R. Schlenk$^*$ and B.
Brors$^*$ and R. B. Russell and H. Glimm$^*$ and M.
Schlesner$^*$ and S. Fröhling$^*$},
title = {{D}efective homologous recombination {DNA} repair as
therapeutic target in advanced chordoma.},
journal = {Nature Communications},
volume = {10},
number = {1},
issn = {2041-1723},
address = {[London]},
publisher = {Nature Publishing Group UK},
reportid = {DKFZ-2019-01097},
pages = {1635},
year = {2019},
abstract = {Chordomas are rare bone tumors with few therapeutic
options. Here we show, using whole-exome and genome
sequencing within a precision oncology program, that
advanced chordomas (n = 11) may be characterized by
genomic patterns indicative of defective homologous
recombination (HR) DNA repair and alterations affecting
HR-related genes, including, for example, deletions and
pathogenic germline variants of BRCA2, NBN, and CHEK2. A
mutational signature associated with HR deficiency was
significantly enriched in $72.7\%$ of samples and
co-occurred with genomic instability. The poly(ADP-ribose)
polymerase (PARP) inhibitor olaparib, which is
preferentially toxic to HR-incompetent cells, led to
prolonged clinical benefit in a patient with refractory
chordoma, and whole-genome analysis at progression revealed
a PARP1 p.T910A mutation predicted to disrupt the
autoinhibitory PARP1 helical domain. These findings uncover
a therapeutic opportunity in chordoma that warrants further
exploration, and provide insight into the mechanisms
underlying PARP inhibitor resistance.},
cin = {A380 / L101 / B080 / A010 / B340 / B240 / B330 / E010 /
A360 / W190 / B290 / L301 / L501 / L701 / L401},
ddc = {500},
cid = {I:(DE-He78)A380-20160331 / I:(DE-He78)L101-20160331 /
I:(DE-He78)B080-20160331 / I:(DE-He78)A010-20160331 /
I:(DE-He78)B340-20160331 / I:(DE-He78)B240-20160331 /
I:(DE-He78)B330-20160331 / I:(DE-He78)E010-20160331 /
I:(DE-He78)A360-20160331 / I:(DE-He78)W190-20160331 /
I:(DE-He78)B290-20160331 / I:(DE-He78)L301-20160331 /
I:(DE-He78)L501-20160331 / I:(DE-He78)L701-20160331 /
I:(DE-He78)L401-20160331},
pnm = {311 - Signalling pathways, cell and tumor biology
(POF3-311)},
pid = {G:(DE-HGF)POF3-311},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:30967556},
pmc = {pmc:PMC6456501},
doi = {10.1038/s41467-019-09633-9},
url = {https://inrepo02.dkfz.de/record/143513},
}