← BioTransfer GEO Dataset Finder
GEO series

AML-PDXs Accelerate Development of Novel Drug Combinations for KMT2A-rearranged Acute Myeloid leukemia

GSE268003 Homo sapiens (human) RNA-seq (gene expression) 31 samples Submitted 2026/04/09 Platform GPL24676
At a glance

RNA-seq (gene expression) · human · 31 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
Homo sapiens (human)
Topic
Acute myeloid leukemiaThis series is part of our curated briefing on it.
Samples
31 samples: 31 samples, each with a distinct title — no replicated groupsGroups 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
Advancing precision therapy in pediatric acute myeloid leukemia through PDX models and mitochondrial targetingBlood advances · 2026
Summary (written by the submitters)
In pediatric acute myeloid leukemia (AML) the achievement of complete remission with standard treatments is obtained in most cases, but relapse still occurs in 30% of patients with greater incidence found in those patients showing high-risk AML genetic features. There is, therefore, an urgent need to identify novel effective therapies for children with relapsed-refractory leukemia.
Read the full summary ↓
We established 26 AML patient-derived xenografts (PDXs), through sequential engraftment in NSG mice, including 14 high-risk genetic subtypes. Results confirmed AML-PDXs robustly resemble the original AML in terms of immunophenotype, genomic, and transcriptomic profiles, offering a comprehensive view of the disease complexity, useful to tailor therapies. In particular, by whole-exome sequencing, we evidenced a high intra-tumoral heterogeneity and we identified variants of each founder clone being perpetuated from patient-AML blasts to PDX. Then, we explored the efficacy of several drugs predicted to target these genomic variants in a three-dimensional in vitro culture and demonstrated that targeting variants of theWnt/β-cateninpathway is a promising strategy to reduce AML subclone fitness. By RNA-sequencing we observed thatKMT2A-rearranged AML and AML-PDXs shared aberrantly activated metabolic and stem expression signatures and documented that both IACS-010759 and Asparaginase, tailored to target transcriptome aberrancies, if combined with Venetoclax, mediated a significantly prolonged PDXs survival compared to Venetoclax used as single agent. Overall, our data indicate that AML-PDX models are unique tools for capturing AML heterogeneity, offering the possibility to target cancer cells in their multiple aspects, this increasing the chance to fully eradicate leukemia cells.
Published in
Advancing precision therapy in pediatric acute myeloid leukemia through PDX models and mitochondrial targeting
Da Ros A, Peloso A, Longo G et al. · Blood advances 2026 · PMID 41576348 · doi:10.1182/bloodadvances.2025018002
This dataset
Open GSE268003 in finder → Find more human RNA-seq datasets → Acute myeloid leukemia landscape →
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 GSE268003_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 PRJNA1114018 and SRA study SRP508966. Searching any of these in the dataset finder brings you back here.

NCBI GEO page ↗ Paper (PMID 41576348) ↗
Study design
31 samples, each with a distinct title — no replicated groups

Any grouping that defines a comparison lives in the sample characteristics, which GEO records separately from the titles.

How we read this: Read from 31 sample titles: 31 distinct titles with little repetition. 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
More Acute myeloid leukemia datasets

Search all human RNA-seq datasets in GEO →

Share this dataset

Metadata from NCBI GEO, cached and refreshed periodically — the NCBI page above is authoritative. Downloads link straight to NCBI/ENA; nothing is proxied through BioTransfer.