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Enhanced antisense oligonucleotide delivery reveals that transcript turnover impacts apparent splicing rescue in myotonic dystrophy

GSE319608 Mus musculus (mouse) RNA-seq (gene expression) 98 samples Submitted 2026/05/28 Platform GPL30172
At a glance

RNA-seq (gene expression) · mouse · 98 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
98 samples: 24 conditions, mostly with about 4 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.
Paper
Enhanced antisense oligonucleotide delivery reveals that transcript turnover impacts apparent splicing rescue in myotonic dystrophyMolecular therapy : the journal of the American Society of Gene Therapy · 2026
Summary (written by the submitters)
Steric-blocking antisense oligonucleotides rescue Myotonic dystrophy type 1 phenotypes in preclinical models and are under evaluation in clinical trials. However, rationale for biomarker selection remains a topic of debate. Here, we show that a cyclic cell-penetrating peptide that escapes endosomes enhances muscle delivery of a phosphorodiamidate morpholino oligonucleotide (PMO) designed to block pathogenic CUG repeat expansions in HSALR mice.
Read the full summary ↓
A single systemic administration rescued mis-splicing and eliminated myotonia one-week post-injection, with partial splicing rescue evident after 24 hours. Interestingly, some exons showed more robust rescue than others, but relationships between MBNL concentration ([MBNL]) and Percent Spliced In (Ψ) could not fully explain extent of rescue. We hypothesized that since pre-existing transcripts must be degraded to reveal full drug effect, rates of transcript replacement might account for these discrepancies. We formulated a mathematical framework and used Bayesian inference to model how apparent Ψ lags behind nascent Ψ as a function of time; faster rates of replacement result in shorter lags. In vivo 5-ethynyl uridine labeling followed by RNAseq validated these predictions. Overall, we show that transcript turnover influences Ψ during periods of dynamically changing [MBNL] and recommend that this be considered when selecting splicing biomarkers and interpreting responses to therapeutic interventions.
Published in
Enhanced antisense oligonucleotide delivery reveals that transcript turnover impacts apparent splicing rescue in myotonic dystrophy
Shea EN, Olafson HR, Muscato DR et al. · Molecular therapy : the journal of the American Society of Gene Therapy 2026 · PMID 42163456 · doi:10.1016/j.ymthe.2026.04.060
This dataset
Open GSE319608 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 GSE319608_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 PRJNA1424719 and SRA study SRP677638. Searching any of these in the dataset finder brings you back here.

NCBI GEO page ↗ Paper (PMID 42163456) ↗
Study design
24 conditions, mostly with about 4 replicates each
HSALR homozygous, Gastrocnemius, PBS, 7 days, ×8 HSALR homozygous, Quadriceps, EEV4-PMO (15 mg… ×6 FVB/NJ, Gastrocnemius, PBS, 7 days, ×4 HSALR homozygous, Gastrocnemius, PMO (30 mg/k… ×4 HSALR homozygous, Gastrocnemius, EEV4-PMO (15… ×4 HSALR homozygous, Gastrocnemius, EEV4-PMO (30… ×4 HSALR homozygous, Gastrocnemius, EEV4-PMO (15… ×4 HSALR homozygous, Gastrocnemius, EEV4-PMO (15… ×4 +16 more

Supports a between-group comparison across 98 samples.

How we read this: 24 replicated groups read from 98 sample titles; they account for 98 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

+ 58 more — browse all 98 samples with per-sample file links →

Datasets on a similar topic

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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.