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Safety and efficacy analysis of in vivo lentiviral gene therapy in pre-clinical ARC syndrome models

GSE329589 Mus musculus (mouse) RNA-seq (gene expression) 10 samples Submitted 2026/05/06 Platform GPL30172
At a glance

RNA-seq (gene expression) · mouse · 10 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
10 samples: 5 conditions, mostly in duplicateGroups 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
Safety and efficacy analysis of in vivo lentiviral gene therapy in pre-clinical ARC syndrome modelsNature communications · 2026
Summary (written by the submitters)
Arthrogryposis, Renal dysfunction and Cholestasis (ARC) syndrome is a rare inherited disorder caused by defects in the VPS33B trafficking protein, leading to impaired bile flow, progressive liver disease and early death. No effective treatments are currently available. Gene therapy offers a potential approach by restoring the missing VPS33B protein in liver cells.
Read the full summary ↓
Here, we show that liver-targeted lentiviral gene therapy safely and effectively rescues key features of ARC syndrome in a mouse model following in vivo administration by intravenous injection, combined with transient liver macrophage depletion. To assess the treatment efficacy, disease severity is exacerbated by 0.25% cholic acid diet feeds. A liver-specific vector shows a favourable safety profile over a ubiquitous vector. Mice receiving the safe liver-specific vector display improved survival, growth and liver function, reduced fibrosis and bile canaliculi restoration. These findings support targeted gene therapy as a promising treatment for ARC syndrome and related early-onset liver diseases.
Published in
Safety and efficacy analysis of in vivo lentiviral gene therapy in pre-clinical ARC syndrome models
Cozmescu CA, Nazari M, Touramanidou L et al. · Nature communications 2026 · PMID 42321174 · doi:10.1038/s41467-026-73631-x
This dataset
Open GSE329589 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 GSE329589_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 PRJNA1460273 and SRA study SRP696248. Searching any of these in the dataset finder brings you back here.

NCBI GEO page ↗ Paper (PMID 42321174) ↗
Study design
5 conditions, mostly in duplicate
Liver, EV-Healthy_8182, biol ×2 Liver, Het-Healthy_1581, biol ×2 Liver, EG-Tumour_1578, biol ×2 Liver, EG-Tumour_3282, biol ×2 Liver, EV-Tumour_8182, biol ×2

Supports a case/control comparison: 6 samples read as cases, 4 as controls.

How we read this: 5 replicated groups read from 10 sample titles; they account for 10 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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