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Delivery of A4GALT targeting siRNA-lipid nanoparticle rescues fabry disease phenotypes in hiPSC derived endothelial cell and podocyte models

GSE282204 Homo sapiens (human) RNA-seq (gene expression) 18 samples Submitted 2025/06/18 Platform GPL21697
At a glance

RNA-seq (gene expression) · human · 18 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
18 samples: 6 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
A4GALT-targeting siRNA lipid nanoparticles ameliorate Fabry disease phenotype: Greater efficacy in endothelial cells than in podocytesMolecular therapy. Nucleic acids · 2025
Summary (written by the submitters)
In this study, we explore the therapeutic feasibility of globotriaosylceramide (Gb3) synthase (A4GALT)-specific siRNA-loaded polyhistidine (pHis)-incorporated lipid nanoparticles (HLNPs) for Fabry disease (FD). HLNPs were developed to deliver siRNAs targeting A4GALT using a microfluidic device, with pHis aiding in endosome escape. The therapy was tested on GLA-knockout human induced pluripotent stem cell-derived endothelial cells (GLA-KO-hiPSC-ECs) and podocytes (GLA-KO-hiPSC-PCs).
Read the full summary ↓
GLA-KO-hiPSCs-ECs or -PCs, upon differentiation, were treated with A4GALT-siRNA-HLNP. Successful intracellular uptake of A4GALT-siRNA-HLNP was confirmed through fluorescence and electron microscopy in both cell types. A4GALT-siRNA-HLNP treatment confirmed both cell types' stability at 5 µg/mL. Increased Gb3 deposition and zebra body formation were detected in both cell types, but A4GALT-siRNA-HLNP treatment attenuated these FD phenotypes, demonstrating reduced expression of A4GALT through western blot analysis. RNA sequencing analysis revealed that the expression of transcripts associated with FD in both cell types was restored by A4GALT-siRNA-HLNP treatment. Suppression of A4GALT via siRNA/HLNP treatment significantly rescued FD phenotypes, presenting a novel therapeutic approach for FD.
Published in
A4GALT-targeting siRNA lipid nanoparticles ameliorate Fabry disease phenotype: Greater efficacy in endothelial cells than in podocytes
Shin YJ, Lee H, Fang X et al. · Molecular therapy. Nucleic acids 2025 · PMID 40520362 · doi:10.1016/j.omtn.2025.102573
This dataset
Open GSE282204 in finder → Find more human 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 GSE282204_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 PRJNA1187579 and SRA study SRP545977. Searching any of these in the dataset finder brings you back here.

NCBI GEO page ↗ Paper (PMID 40520362) ↗
Study design
6 conditions, mostly in triplicate
GLA-KO hiPSC-EC ×3 GLA-KO hiPSC-PC ×3 GLA-KO+siRNA-HLNP hiPSC-EC ×3 GLA-KO+siRNA-HLNP hiPSC-PC ×3 WTC11 hiPSC-EC ×3 WTC11 hiPSC-PC ×3

Supports a between-group comparison across 18 samples.

How we read this: 6 replicated groups read from 18 sample titles; they account for 18 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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