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@ARTICLE{Okonechnikov:300822,
author = {K. Okonechnikov$^*$ and P. K. Joshi$^*$ and V. Körber$^*$
and A. Rademacher$^*$ and M. Bortolomeazzi$^*$ and J.-P.
Mallm$^*$ and J. Vaillant$^*$ and P. B. G. da Silva$^*$ and
B. Statz$^*$ and M. Sepp and I. Sarropoulos and T. Yamada
and A. Wittmann$^*$ and K. Schramm$^*$ and M.
Blattner-Johnson$^*$ and P. Fiesel$^*$ and B. Jones$^*$ and
N. Jäger$^*$ and T. Milde$^*$ and K. Pajtler$^*$ and C. M.
van Tilburg$^*$ and O. Witt$^*$ and K. Bochennek and K. J.
Weber$^*$ and L. Nonnenmacher and C. Reimann and D. R.
Ghasemi$^*$ and U. Schüller and M. Mynarek and S. Rutkowski
and D. Jones$^*$ and A. Korshunov$^*$ and K. Rippe$^*$ and
F. Westermann$^*$ and S. Thongjuea$^*$ and T. Höfer$^*$ and
H. Kaessmann$^*$ and L. Kutscher$^*$ and S. Pfister$^*$},
title = {{O}ncogene aberrations drive medulloblastoma progression,
not initiation.},
journal = {Nature},
volume = {642},
number = {8069},
issn = {0028-0836},
address = {London [u.a.]},
publisher = {Nature Publ. Group},
reportid = {DKFZ-2025-00936},
pages = {1062-1072},
year = {2025},
note = {DKFZ-ZMBH Alliance /
#EA:B062#EA:B430#EA:B086#LA:B430#LA:B062# / 2025
Jun;642(8069):1062-1072},
abstract = {Despite recent advances in understanding disease biology,
treatment of group 3/4 medulloblastoma remains a therapeutic
challenge in paediatric neuro-oncology1. Bulk-omics
approaches have identified considerable intertumoural
heterogeneity in group 3/4 medulloblastoma, including the
presence of clear single-gene oncogenic drivers in only a
subset of cases, whereas in most cases, large-scale copy
number aberrations prevail2,3. However, intratumoural
heterogeneity, the role of oncogene aberrations, and broad
copy number variation in tumour evolution and treatment
resistance remain poorly understood. To dissect this
interplay, we used single-cell technologies (single-nucleus
RNA sequencing (snRNA-seq), single-nucleus assay for
transposase-accessible chromatin with high-throughput
sequencing (snATAC-seq) and spatial transcriptomics) on a
cohort of group 3/4 medulloblastoma with known alterations
in the oncogenes MYC, MYCN and PRDM6. We show that
large-scale chromosomal aberrations are early
tumour-initiating events, whereas the single-gene oncogenic
events arise late and are typically subclonal, but MYC can
become clonal upon disease progression to drive further
tumour development and therapy resistance. Spatial
transcriptomics shows that the subclones are mostly
interspersed across tumour tissue, but clear segregation is
also present. Using a population genetics model, we estimate
medulloblastoma initiation in the cerebellar unipolar brush
cell lineage starting from the first gestational trimester.
Our findings demonstrate how single-cell technologies can be
applied for early detection and diagnosis of this fatal
disease.},
cin = {B062 / HD01 / B430 / B086 / B066 / W192 / B360 / B300 /
B310 / FM01 / B087},
ddc = {500},
cid = {I:(DE-He78)B062-20160331 / I:(DE-He78)HD01-20160331 /
I:(DE-He78)B430-20160331 / I:(DE-He78)B086-20160331 /
I:(DE-He78)B066-20160331 / I:(DE-He78)W192-20160331 /
I:(DE-He78)B360-20160331 / I:(DE-He78)B300-20160331 /
I:(DE-He78)B310-20160331 / I:(DE-He78)FM01-20160331 /
I:(DE-He78)B087-20160331},
pnm = {312 - Funktionelle und strukturelle Genomforschung
(POF4-312)},
pid = {G:(DE-HGF)POF4-312},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:40335697},
doi = {10.1038/s41586-025-08973-5},
url = {https://inrepo02.dkfz.de/record/300822},
}