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@ARTICLE{Lepp:294687,
author = {A.-M. Leppä$^*$ and K. Grimes and H. Jeong and F. Y.
Huang$^*$ and A. Andrades and A. Waclawiczek$^*$ and T. Boch
and A. Jauch and S. Renders$^*$ and P. Stelmach$^*$ and C.
Müller-Tidow and D. Karpova$^*$ and M. Sohn$^*$ and F.
Grünschläger$^*$ and P. Hasenfeld and E. Benito Garagorri
and V. Thiel$^*$ and A. Dolnik and B. Rodriguez-Martin and
L. Bullinger and K. Mrózek and A.-K. Eisfeld and A.
Krämer$^*$ and A. D. Sanders and J. Korbel$^*$ and A.
Trumpp$^*$},
title = {{S}ingle-cell multiomics analysis reveals dynamic clonal
evolution and targetable phenotypes in acute myeloid
leukemia with complex karyotype.},
journal = {Nature genetics},
volume = {56},
number = {12},
issn = {1061-4036},
address = {London},
publisher = {Macmillan Publishers Limited, part of Springer Nature},
reportid = {DKFZ-2024-02422},
pages = {2790-2803},
year = {2024},
note = {DKFZ-ZMBH Alliance / #EA:A010#LA:A010#LA:B480# / 2024
Dec;56(12):2790-2803},
abstract = {Chromosomal instability is a major driver of intratumoral
heterogeneity (ITH), promoting tumor progression. In the
present study, we combined structural variant discovery and
nucleosome occupancy profiling with transcriptomic and
immunophenotypic changes in single cells to study ITH in
complex karyotype acute myeloid leukemia (CK-AML). We
observed complex structural variant landscapes within
individual cells of patients with CK-AML characterized by
linear and circular breakage-fusion-bridge cycles and
chromothripsis. We identified three clonal evolution
patterns in diagnosis or salvage CK-AML (monoclonal, linear
and branched polyclonal), with $75\%$ harboring multiple
subclones that frequently displayed ongoing karyotype
remodeling. Using patient-derived xenografts, we
demonstrated varied clonal evolution of leukemic stem cells
(LSCs) and further dissected subclone-specific drug-response
profiles to identify LSC-targeting therapies, including
BCL-xL inhibition. In paired longitudinal patient samples,
we further revealed genetic evolution and cell-type
plasticity as mechanisms of disease progression. By
dissecting dynamic genomic, phenotypic and functional
complexity of CK-AML, our findings offer clinically relevant
avenues for characterizing and targeting disease-driving
LSCs.},
cin = {A010 / A360 / B480 / HD01},
ddc = {570},
cid = {I:(DE-He78)A010-20160331 / I:(DE-He78)A360-20160331 /
I:(DE-He78)B480-20160331 / I:(DE-He78)HD01-20160331},
pnm = {311 - Zellbiologie und Tumorbiologie (POF4-311)},
pid = {G:(DE-HGF)POF4-311},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:39587361},
doi = {10.1038/s41588-024-01999-x},
url = {https://inrepo02.dkfz.de/record/294687},
}