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Anti-asthma drug Montelukast induces autistic behaviors via disrupting neuronal retinoic acid signaling [RNA-seq and Ribo-seq]

GSE232669 Homo sapiens (human) RNA-seq (gene expression) 18 samples Submitted 2026/03/16 Platform GPL16791
At a glance

RNA-seq (gene expression) · human · 18 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)
Samples
18 samples: 6 conditions, mostly in triplicateGroups 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
Anti-asthma drug montelukast induces autistic behaviors via disrupting neuronal retinoic acid signalingSignal transduction and targeted therapy · 2026
Summary (written by the submitters)
Globally, autism spectrum disorder (ASD) affects approximately 1.0-2.5% of human newborns, with non-genetic risk factors estimated to contribute to over 50% ASD cases through yet elusive mechanisms. Montelukast (MTK), a leukotriene receptor antagonist and FDA-approved anti-asthma drug, was found to potently disrupt retinoic acid (RA) signaling, and substantially increase the risk of ASD in humans when used during pregnancy or in early childhood, according to prospective and retrospective cohort studies.
Read the full summary ↓
Both maternal and postnatal exposure to montelukast caused autistic-like behaviors in rat offspring. Montelukast also altered brain regional patterning through disrupting RA signaling in human cerebral organoids, similar to DEAB, an established inhibitor of RA-synthesizing ALDH1As. Mechanistically, montelukast was found to directly bind to retinoic acid receptor alpha (RARα) and impair RARα heterodimer formation to disrupt RA signaling. Altogether, this work highlighted not only the developmental origin of ASD, but the utmost importance in cautioned use of medicinal chemicals during maternity or early childhood to prevent psychiatric disorders, e.g. ASD.
Published in
Anti-asthma drug montelukast induces autistic behaviors via disrupting neuronal retinoic acid signaling
Hao ZJ, Wu QH, Li YL et al. · Signal transduction and targeted therapy 2026 · PMID 42230569 · doi:10.1038/s41392-026-02665-w
This dataset
Open GSE232669 in finder → Find more human 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 GSE232669_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 PRJNA973551 and SRA study SRP438172. Searching any of these in the dataset finder brings you back here.

NCBI GEO page ↗ Paper (PMID 42230569) ↗
Study design
6 conditions, mostly in triplicate
Brain Organoid, DMSO, Day 60, RNAseq, biol ×3 Brain Organoid, DEAB, Day 60, RNAseq, biol ×3 Brain Organoid, MTK, Day 60, RNAseq, biol ×3 Brain Organoid, DMSO, Day 60, Riboseq, biol ×3 Brain Organoid, DEAB, Day 60, Riboseq, biol ×3 Brain Organoid, MTK, Day 60, Riboseq, biol ×3

Supports a between-group comparison across 18 samples.

How we read this: 6 replicated groups read from 18 sample titles; they account for 18 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
Datasets on a similar topic

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