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The drug-elicitable alternative splicing module for tunable vector expression in the heart

GSE270445 Homo sapiens (human) RNA-seq (gene expression) 36 samples Submitted 2025/04/13 Platform GPL29480Platform GPL24247Platform GPL28330
At a glance

RNA-seq (gene expression) · human · 36 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
36 samples: 10 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
The drug-elicitable alternative splicing module for tunable vector expression in the heartNature cardiovascular research · 2025
Summary (written by the submitters)
RNA Seq datasets from WT or AAV-injected mice treated with 10 mg/kg risdiplam or solvent. RNA Seq datasets from plasmid-transfection HEK293T cells treated with 1uM risdiplam or DMSO.
Published in
The drug-elicitable alternative splicing module for tunable vector expression in the heart
Chen Z, Yang L, Zhang Y et al. · Nature cardiovascular research 2025 · PMID 40514436 · doi:10.1038/s44161-025-00665-7
This dataset
Open GSE270445 in finder → Find more 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 GSE270445_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 PRJNA1126649 and SRA study SRP515245. Searching any of these in the dataset finder brings you back here.

NCBI GEO page ↗ Paper (PMID 40514436) ↗
Study design
10 conditions, mostly in triplicate
WT, risdiplam, 1 day post risdiplam, ×5 WT, solvent, 1 day post solvent, ×5 AAV-injection, risdiplam, 30 day post risdipl… ×4 AAV-injection, solvent, 30 day post solvent, ×4 AAV-injection, solvent, 1 day post solvent, ×3 AAV-injection, risdiplam,1 day post risdiplam, ×3 AAV-injection, solvent, 11 days post solvent, ×3 AAV-injection, risdiplam,11 days post risdipl… ×3 +2 more

Supports a case/control comparison: 3 samples read as cases, 10 as controls.

How we read this: 10 replicated groups read from 36 sample titles; they account for 36 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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