MYST acetyltransferases are a targetable therapeutic vulnerability in SETBP1-mutant leukemia

SETBP1 mutations are markers of high-risk myeloid malignancies, but we lack any targeted therapies to improve outcomes. In this study, we identify MYST acetyltransferases as key drivers of mutant SETBP1-driven transcription. MYST inhibitors are highly effective against SETBP1-mutant leukemia and represent a promising avenue for clinical translation.

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Publication Details

Journal
bioRxiv (Cold Spring Harbor Laboratory)
Published
2026-01-08
DOI
https://doi.org/10.64898/2026.01.08.697228
Primary Topic
Acute Myeloid Leukemia Research
Type
article
Field-Weighted Citation Impact
0.00
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article

MYST acetyltransferases are a targetable therapeutic vulnerability in SETBP1-mutant leukemia

bioRxiv (Cold Spring Harbor Laboratory)
Acute Myeloid Leukemia Research
article

MYST acetyltransferases are a targetable therapeutic vulnerability in SETBP1-mutant leukemia

article en

Abstract

SETBP1 mutations are markers of high-risk myeloid malignancies, but we lack any targeted therapies to improve outcomes. In this study, we identify MYST acetyltransferases as key drivers of mutant SETBP1-driven transcription. MYST inhibitors are highly effective against SETBP1-mutant leukemia and represent a promising avenue for clinical translation.

bioRxiv (Cold Spring Harbor Laboratory)
Pacific Northwest National Laboratory (US), Oregon Health & Science University (US), Moffitt Cancer Center (US), OHSU Knight Cancer Institute
Good health and well-being
Openalex Percentile: Top 94%
Acute Myeloid Leukemia Research
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