GEO series

Bottlebrush Polymer Conjugates for Enhanced Antisense Oligonucleotide Therapy in Myotonic Dystrophy Type 1

GSE309640 Mus musculus (mouse) RNA-seq (gene expression) 30 samples Submitted 2026/07/01 Platform GPL24247

At a glance

RNA-seq (gene expression) · mouse · 30 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: 30 samples: 19 distinct titles, 2 of them repeatedGroups 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: Bottlebrush polymer conjugates for enhanced antisense oligonucleotide therapy in myotonic dystrophy type 1Nucleic acids research · 2026

Summary (written by the submitters)

Oligonucleotides are a promising class of genetic medicine for myotonic dystrophy type 1 (DM1), the most common adult-onset muscular dystrophy. However, poor muscle distribution of nucleic acid drugs following systemic administration has hindered drug development, and no curative treatment currently exists. DM1 pathology requires drug localization to the nucleus, where pathogenic mutant RNA is sequestered, posing additional challenges after cellular internalization regarding endosomal escape and nuclear uptake.

Read the full summary ↓

Here, we show that a locked nucleic acid oligonucleotide targeting mutant CUG repeat RNA tracts, conjugated to a bottlebrush polymer, exhibits improved muscle distribution and potent correction of DM1-associated splicing dysregulation in a DM1 mouse model. Significant improvements in myotonia, body weight, and grip strength were observed. The conjugate was well tolerated following 12 weeks of weekly intravenous administration. These findings suggest that bottlebrush polymer conjugates may overcome key limitations of conventional oligonucleotide therapeutics for neuromuscular conditions, with potential to become a potent and cost-effective DM1 therapy.

Published in

Bottlebrush polymer conjugates for enhanced antisense oligonucleotide therapy in myotonic dystrophy type 1

Li Y, Oetheimer C, Wang Y et al. · Nucleic acids research 2026 · PMID 42339623 · doi:10.1093/nar/gkag619

This dataset

Open GSE309640 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 GSE309640_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 PRJNA1336658 and SRA study SRP630036. Searching any of these in the dataset finder brings you back here.

NCBI GEO page ↗ Paper (PMID 42339623) ↗

Study design

19 distinct titles, 2 of them repeated

NT ×8 WT ×5

How we read this: Read from 30 sample titles: 19 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

GSM9271013 WT1
GSM9271014 WT2
GSM9271015 WT3
GSM9271016 WT4
GSM9271017 WT5
GSM9271018 NT1
GSM9271019 NT2
GSM9271020 NT3
GSM9271021 NT4
GSM9271022 NT5
GSM9271023 NT6
GSM9271024 pacDNA-L9 1A
GSM9271025 pacDNA-L9 1B
GSM9271026 pacDNA-L9 4A
GSM9271027 pacDNA-L9 4B
GSM9271028 pacDNA-L9 8A
GSM9271029 pacDNA-L9 8B
GSM9271030 pacDNA-L9 8C
GSM9271031 Brush A
GSM9271032 Brush B
GSM9271033 pacDNA-Scr A
GSM9271034 pacDNA-Scr B
GSM9271035 L9 A
GSM9271036 L9 B
GSM9708445 NT7
GSM9708446 NT8
GSM9708447 pacDNA-L9 1C
GSM9708448 pacDNA-L9 4C
GSM9708449 L9 C
GSM9708450 pacDNA-L9 1D

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