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MYST acetyltransferases are a targetable therapeutic vulnerability in SETBP1-mutant leukemia [RNA-Seq]

GSE303625 Mus musculus (mouse) RNA-seq (gene expression) 14 samples Submitted 2026/04/01 Platform GPL30172Platform GPL34290
At a glance

RNA-seq (gene expression) · mouse · 14 samples

What it is
RNA-seq (gene expression)Gene activity measured by sequencing. The summary does not mention single-cell work, so this is most likely bulk: one average per sample, the usual starting point for comparing groups.
Organism
Mus musculus (mouse)
Samples
14 samples: 4 × Lin- cells, DMSO control, vs 4 × Lin- cells, PF9363 treatment, vs 3 × Lin- cells, Luciferase control, vs 3 × Lin- cells, SETBP1D868N mutant,Groups are read from the sample names, not from the paper. Check the list below.
Data you can get
A processed table (series matrix) and supplementary files from NCBI; the raw sequencing reads from ENA. Which of these hold counts depends on the study, so check the file list.
Paper
MYST Acetyltransferases Interact with SETBP1 and Are a Targetable Therapeutic Vulnerability in SETBP1-Mutant LeukemiaBlood cancer discovery · 2026
Summary (written by the submitters)
Mutations in SET binding protein 1 (SETBP1) are associated with an adverse prognosis in myeloid malignancies. These mutations stabilize SETBP1 protein, driving increased expression of a progenitor-associated gene expression program through incompletely described mechanisms. A proteomic screen revealed interactions between SETBP1 and members of the MYST acetyltransferase complexes, including the catalytic subunits—KAT6A and KAT7.
Read the full summary ↓
Mutant SETBP1 increases the localization of MYST complexes at known SETBP1 target genes, including the Hoxa cluster, where they drive increased histone acetylation and gene expression. Treatment of SETBP1D868N-expressing myeloid progenitors with MYST inhibitors reduced target gene expression. To establish the efficacy of MYST inhibition in vivo, we treated mice harboring a syngeneic SETBP1-mutant leukemia with the clinical-grade MYST inhibitor—PF-9363. This resulted in complete hematologic control and increased survival. MYST inhibition was also highly effective against a SETBP1-mutant PDX model. These studies identify MYST acetyltransferases as promising therapeutic targets in SETBP1-mutant malignancies.
Published in
MYST Acetyltransferases Interact with SETBP1 and Are a Targetable Therapeutic Vulnerability in SETBP1-Mutant Leukemia
Carlson HL, Nguyen TT, Tauchmann S et al. · Blood cancer discovery 2026 · PMID 42001499 · doi:10.1158/2643-3230.BCD-25-0446
This dataset
Open GSE303625 in finder → Find more mouse RNA-seq datasets →
Download · which file do I need?

Direct links to NCBI and ENA, no account and no request form. Pick by what you want to do: take the small processed table to look, the supplementary files to re-analyse, or the original raw data to start over.

1 · Just have a look

Processed table

The series matrix: one table of values per sample as the submitters processed it. The smallest download, and it opens in Excel or R. Start here to see what the data look like.

Series matrix ↓
2 · Re-analyse it

Supplementary files

The count tables and other files the authors uploaded. Take only the samples you need, or the whole study as GSE303625_RAW.tar (it can run to tens of gigabytes).

All supplementary files ↗ Whole study · RAW.tar ↓
3 · Start from the raw reads

Raw sequencing reads

The original FASTQ files, straight from the sequencer, held at ENA. Large, and you align them yourself, so only choose this if you need to re-process the study from scratch.

Raw reads on ENA ↗

What the files usually look like: For RNA-seq the counts are usually a table among the supplementary files (look for 'counts', 'expression', 'FPKM' or 'TPM' in the file name). The series matrix of a sequencing study is sometimes nearly empty.

Also filed as BioProject PRJNA1295953 and SRA study SRP603301. Searching any of these in the dataset finder brings you back here.

NCBI GEO page ↗ Paper (PMID 42001499) ↗
Study design
4 × Lin- cells, DMSO control, vs 4 × Lin- cells, PF9363 treatment, vs 3 × Lin- cells, Luciferase control, vs 3 × Lin- cells, SETBP1D868N mutant,

Supports a case/control comparison: 3 samples read as cases, 7 as controls.

How we read this: 4 replicated groups read from 14 sample titles; they account for 14 of them. These groups are guessed from the sample names, not taken from the paper, so check them against the sample list below before relying on them.

Samples in this study
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