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@ARTICLE{Khn:301475,
author = {M. W. M. Kühn$^*$ and N. Pemmaraju and F. H. Heidel},
title = {{T}he evolving landscape of epigenetic target molecules and
therapies in myeloid cancers: focus on acute myeloid
leukemia and myeloproliferative neoplasms.},
journal = {Leukemia},
volume = {39},
number = {8},
issn = {0887-6924},
address = {London},
publisher = {Springer Nature},
reportid = {DKFZ-2025-01017},
pages = {1824-1837},
year = {2025},
note = {39(8):1824-1837},
abstract = {Research on myeloid neoplasms, a field that has been
driving scientific advances in cancer for over 50 years, has
yielded many discoveries that have fundamentally reshaped
our understanding of cancer biology. These insights, often
the product of leukemia research, have been instrumental in
developing more mechanism-based treatments in the early
2000s [1]. Recognizing epigenetic dysregulation as a common
disease mechanism in myeloid cancers has been groundbreaking
regarding recent treatment developments that exploit
chromatin-based oncogenic mechanisms. In the case of acute
myeloid leukemia (AML), sequencing studies aimed at
assessing the complement of genetic alterations demonstrated
that more than $60\%$ of AML cases harbored disease-driving
mutations in epigenetic regulators. This high prevalence
underscores the importance of epigenetic dysregulation in
AML pathogenesis [2, 3]. Chromatin regulators commonly
control disease-specific transcriptional programs, making
them attractive therapeutic targets to manipulate neoplastic
gene expression programs, particularly in myeloid neoplasms.
Several drugs targeting epigenetic mechanisms and exploiting
myeloid disease-specific dependencies have recently been
approved for treating myeloid neoplasms. Many additional
drugs are currently being investigated in clinical trials,
and numerous new compound developments are being studied in
preclinical studies. This manuscript will review (1)
chromatin-based disease mechanisms, such as DNA methylation,
chromatin regulatory complexes, and histone modifications,
currently investigated for therapeutic exploitation in
myeloid malignancies, and (2) therapeutic developments
already approved or investigated for treating these
diseases.},
subtyp = {Review Article},
cin = {FM01},
ddc = {610},
cid = {I:(DE-He78)FM01-20160331},
pnm = {899 - ohne Topic (POF4-899)},
pid = {G:(DE-HGF)POF4-899},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:40374809},
doi = {10.1038/s41375-025-02639-x},
url = {https://inrepo02.dkfz.de/record/301475},
}