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@ARTICLE{Vaillant:127681,
author = {C. Vaillant and P. Valdivieso and S. Nuciforo and M.
Kool$^*$ and A. Schwarzentruber-Schauerte and H. Méreau and
E. Cabuy and J. A. Lobrinus and S. Pfister$^*$ and A. Zuniga
and S. Frank and R. Zeller},
title = {{S}erpine2/{PN}-1 {I}s {R}equired for {P}roliferative
{E}xpansion of {P}re-{N}eoplastic {L}esions and {M}alignant
{P}rogression to {M}edulloblastoma.},
journal = {PLoS one},
volume = {10},
number = {4},
issn = {1932-6203},
address = {Lawrence, Kan.},
publisher = {PLoS},
reportid = {DKFZ-2017-03704},
pages = {e0124870 -},
year = {2015},
abstract = {Medulloblastomas are malignant childhood brain tumors that
arise due to the aberrant activity of developmental pathways
during postnatal cerebellar development and in adult humans.
Transcriptome analysis has identified four major
medulloblastoma subgroups. One of them, the Sonic hedgehog
(SHH) subgroup, is caused by aberrant Hedgehog signal
transduction due to mutations in the Patched1 (PTCH1)
receptor or downstream effectors. Mice carrying a Patched-1
null allele (Ptch1∆/+) are a good model to study the
alterations underlying medulloblastoma development as a
consequence of aberrant Hedgehog pathway
activity.Transcriptome analysis of human medulloblastomas
shows that SERPINE2, also called Protease Nexin-1 (PN-1) is
overexpressed in most medulloblastomas, in particular in the
SHH and WNT subgroups. As siRNA-mediated lowering of
SERPINE2/PN-1 in human medulloblastoma DAOY cells reduces
cell proliferation, we analyzed its potential involvement in
medulloblastoma development using the Ptch1∆/+ mouse
model. In Ptch1∆/+ mice, medulloblastomas arise as a
consequence of aberrant Hedgehog pathway activity. Genetic
reduction of Serpine2/Pn-1 interferes with medulloblastoma
development in Ptch1∆/+ mice, as $~60\%$ of the
pre-neoplastic lesions (PNLs) fail to develop into
medulloblastomas and remain as small cerebellar nodules. In
particular the transcription factor Atoh1, whose expression
is essential for development of SHH subgroup
medulloblastomas is lost. Comparative molecular analysis
reveals the distinct nature of the PNLs in young
Ptch1∆/+Pn-1Δ/+ mice. The remaining wild-type Ptch1
allele escapes transcriptional silencing in most cases and
the aberrant Hedgehog pathway activity is normalized.
Furthermore, cell proliferation and the expression of the
cell-cycle regulators Mycn and Cdk6 are significantly
reduced in PNLs of Ptch1∆/+Pn-1Δ/+ mice.Our analysis
provides genetic evidence that aberrant Serpine2/Pn-1 is
required for proliferation of human and mouse
medulloblastoma cells. In summary, our analysis shows that
Serpine2/PN-1 boosts malignant progression of PNLs to
medulloblastomas, in which the Hedgehog pathway is activated
in a SHH ligand-independent manner.},
keywords = {Hedgehog Proteins (NLM Chemicals) / PTCH protein, human
(NLM Chemicals) / Patched Receptors (NLM Chemicals) /
Patched-1 Receptor (NLM Chemicals) / Ptch1 protein, mouse
(NLM Chemicals) / Receptors, Cell Surface (NLM Chemicals) /
Serpin E2 (NLM Chemicals) / Matrix Metalloproteinase 9 (NLM
Chemicals)},
cin = {B062},
ddc = {500},
cid = {I:(DE-He78)B062-20160331},
pnm = {312 - Functional and structural genomics (POF3-312)},
pid = {G:(DE-HGF)POF3-312},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:25901736},
pmc = {pmc:PMC4406471},
doi = {10.1371/journal.pone.0124870},
url = {https://inrepo02.dkfz.de/record/127681},
}