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Microdystrophins Partially Rescue Duchenne Muscular Dystrophy Deficits in iPSC-Cardiomyocytes

GSE310327 Homo sapiens (human) RNA-seq (gene expression) 34 samples Submitted 2026/06/30 Platform GPL34284
At a glance

RNA-seq (gene expression) · human · 34 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
34 samples: 8 conditions, mostly with about 5 replicates eachGroups 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.
Summary (written by the submitters)
Duchenne muscular dystrophy (DMD) is a severe muscle wasting disease caused by the lack of dystrophin. Dilated cardiomyopathy is the leading cause of death in DMD patients. Smaller variants of dystrophin, called microdystrophins, amenable to packaging into adeno-associated virus (AAV), have shown to be effective in improving skeletal muscle function in animal models; however, the functional benefit of these microdystrophins in the DMD heart remains unclear.
Read the full summary ↓
To determine the efficacy of microdystrophin gene therapy, we compared three microdystrophin variants in DMD cardiomyocytes differentiated from human induced pluripotent stem cells (iPSCs). We used three DMD lines of different genetic backgrounds and benchmarked against healthy isogenic controls expressing full-length dystrophin. DMD iPSC-cardiomyocytes exhibit hallmarks of disease, including arrhythmia and poor viability compared to healthy isogenic controls. In parallel, we tested a dystrophin variant, called minidystrophin, which is larger than the microdystrophins. We validated dystrophin expression and compared transcriptional profiles by bulk RNA sequencing. For morphological analysis, we performed immunohistochemistry of cardiac troponin on fixed cells. To test the functional benefit of the transgenes, we performed calcium imaging with a ratiometric calcium indicator as well as viability assays. Our results show that the microdystrophins partially rescue disease phenotypes; however, the results are variable among genetic backgrounds. None of the gene therapies altered the global transcriptional profile of the DMD iPSC-CMs to resemble a healthy profile. Minidystrophin significantly improved cell viability of DMD iPSC-CMs in two of three lines of different genetic backgrounds. Our findings suggest that microdystrophins may have limited efficacy in DMD cardiomyocytes and that the delivery of larger dystrophin variants may be necessary to delay the onset of cardiac complications.
This dataset
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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 GSE310327_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 PRJNA1365766 and SRA study SRP647765. Searching any of these in the dataset finder brings you back here.

NCBI GEO page ↗
Study design
8 conditions, mostly with about 5 replicates each
DMD19 iso Day 30 iPSC-CMs treated with empty … ×5 DMD19 Day 30 iPSC-CMs treated with microDYS1,… ×5 DMD19 Day 30 iPSC-CMs treated with microDYS2,… ×5 DMD19 Day 30 iPSC-CMs treated with microDYS3,… ×5 DMD19 Day 30 iPSC-CMs treated with empty vect… ×4 DMD19 Day 30 iPSC-CMs treated with miniDYS, b… ×4 DMD19 iPSCs, untreated, biol ×3 DMD19 iso iPSCs, untreated, biol ×3

Supports a between-group comparison across 34 samples.

How we read this: 8 replicated groups read from 34 sample titles; they account for 34 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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