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Unraveling Novel Drug candidate for ALS through an Integrative Network Analysis of approved Drug-Gene Expression Signatures in Human Pluripotent Stem Cell-Based Model

GSE261648 Homo sapiens (human) RNA-seq (gene expression) 151 samples Submitted 2026/06/22 Platform GPL11154
At a glance

RNA-seq (gene expression) · human · 151 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
151 samples: 51 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.
Summary (written by the submitters)
Given the high attrition rates, substantial costs and slow pace of new drug discovery and development, repurposing of 'old' drugs to treat both common and rare diseases is increasingly becoming an attractive proposition. In this study, we evaluated the potential of using human pluripotent stem cells to identify repurposable drug candidates.
Read the full summary ↓
For this purpose, we treated derivatives from human embryonic stem cells with 50 marketed drugs and we annotated the induced molecular changes by RNA deep sequencing. Focusing on genes previously implicated in monogenic diseases, we identified drugs capable of modulating the expression of PLP1, PMP22, LMNB1 and SQSTM1 genes known to be involved in Pelizaeus-Merzbacher disease (PMP), hereditary neuropathy with liability to pressure palsies (HNPP), adult-onset autosomal dominant leukodystrophy (ALDL) and amyotrophic lateral sclerosis (ALS).
This dataset
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.

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 GSE261648_RAW.tar (it can run to tens of gigabytes).

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.

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 PRJNA1088159 and SRA study SRP495345. Searching any of these in the dataset finder brings you back here.

Study design
51 conditions, mostly in triplicate
DMSO, ×4 Amoxa, ×3 Argat, ×3 ChloDipho, ×3 CyprHydro, ×3 Dexme, ×3 DivaSodium, ×3 EsomMagne, ×3 +43 more

Supports a between-group comparison across 151 samples.

How we read this: 51 replicated groups read from 151 sample titles; they account for 151 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

+ 111 more — browse all 151 samples with per-sample file links →

Datasets on a similar topic

Search all human RNA-seq datasets in GEO →

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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.