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@ARTICLE{Pastoors:290235,
author = {D. Pastoors and M. Havermans and R. Mulet-Lazaro and D.
Brian and W. Noort and J. Gräsel$^*$ and R. Hoogenboezem
and L. Smeenk and J. A. A. Demmers and M. Milsom$^*$ and T.
Enver and R. W. J. Groen and E. Bindels and R. Delwel},
title = {{O}ncogene {EVI}1 drives acute myeloid leukemia via a
targetable interaction with {CTBP}2.},
journal = {Science advances},
volume = {10},
number = {20},
issn = {2375-2548},
address = {Washington, DC [u.a.]},
publisher = {Assoc.},
reportid = {DKFZ-2024-01051},
pages = {eadk9076},
year = {2024},
abstract = {Acute myeloid leukemia (AML) driven by the activation of
EVI1 due to chromosome 3q26/MECOM rearrangements is
incurable. Because transcription factors such as EVI1 are
notoriously hard to target, insight into the mechanism by
which EVI1 drives myeloid transformation could provide
alternative avenues for therapy. Applying protein folding
predictions combined with proteomics technologies, we
demonstrate that interaction of EVI1 with CTBP1 and CTBP2
via a single PLDLS motif is indispensable for leukemic
transformation. A 4× PLDLS repeat construct outcompetes
binding of EVI1 to CTBP1 and CTBP2 and inhibits
proliferation of 3q26/MECOM rearranged AML in vitro and in
xenotransplant models. This proof-of-concept study opens the
possibility to target one of the most incurable forms of AML
with specific EVI1-CTBP inhibitors. This has important
implications for other tumor types with aberrant expression
of EVI1 and for cancers transformed by different
CTBP-dependent oncogenic transcription factors.},
keywords = {Leukemia, Myeloid, Acute: genetics / Leukemia, Myeloid,
Acute: metabolism / Leukemia, Myeloid, Acute: pathology /
MDS1 and EVI1 Complex Locus Protein: metabolism / MDS1 and
EVI1 Complex Locus Protein: genetics / Alcohol
Oxidoreductases: metabolism / Alcohol Oxidoreductases:
genetics / Humans / Animals / DNA-Binding Proteins:
metabolism / DNA-Binding Proteins: genetics / Mice /
Co-Repressor Proteins: metabolism / Co-Repressor Proteins:
genetics / Protein Binding / Cell Line, Tumor / Cell
Proliferation / Cell Transformation, Neoplastic: genetics /
Cell Transformation, Neoplastic: metabolism / Transcription
Factors: metabolism / Transcription Factors: genetics},
cin = {A012},
ddc = {500},
cid = {I:(DE-He78)A012-20160331},
pnm = {311 - Zellbiologie und Tumorbiologie (POF4-311)},
pid = {G:(DE-HGF)POF4-311},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:38748792},
doi = {10.1126/sciadv.adk9076},
url = {https://inrepo02.dkfz.de/record/290235},
}