← BioTransfer GEO Dataset Finder
GEO series

Repurposing niclosamide in mouse models of amyotrophic lateral sclerosis

GSE249071 Mus musculus (mouse) RNA-seq (gene expression) 14 samples Submitted 2024/05/09 Platform GPL24247
At a glance

RNA-seq (gene expression) · mouse · 14 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
14 samples: 4 × spinal cord, veh end stage, vs 4 × spinal cord, nic end stage, vs 3 × spinal cord, veh symptomatic, vs 3 × spinal cord, nic symptomatic,Groups 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
Neuroprotective effects of niclosamide on disease progression via inflammatory pathways modulation in SOD1-G93A and FUS-associated amyotrophic lateral sclerosis modelsNeurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2024
Summary (written by the submitters)
ALS is a complex neurodegenerative disease influenced by genetic, epigenetic, and environmental factors, resulting in dysfunction in cellular and molecular pathways. The limited efficacy of current treatments highlights the need for combination therapies targeting multiple aspects of the disease. Niclosamide, an anthelminthic drug listed as an essential medicine, has been repurposed in clinical trials due to its anti-inflammatory and anti-fibrotic properties.
Read the full summary ↓
Niclosamide can inhibit various molecular pathways (such as STAT3 and mTOR) that are dysregulated in ALS, suggesting its potential to disrupt these altered mechanisms associated with the pathology. Our recent findings have demonstrated that niclosamide can effectively inhibit inflammatory and fibrotic molecular pathways in ALS models. We administered niclosamide intraperitoneally to two transgenic murine models, FUS and SOD1-G93A mice, which replicate key pathologies and biological processes of ALS. The treatment was initiated at the onset of symptoms, and we assessed the progression of the disease by neurological scores, rotarod and grip tests, as well as monitoring survival. Additionally, we examined the effects of the treatment on spinal cord and muscle degeneration in the treated mice. In both models, the administration of niclosamide resulted in a slowdown of disease progression, an increase in survival rates, and an improvement in tissue pathology. This was characterized by a reduction in gliosis, motor neuron loss, muscle atrophy, and inflammatory markers. Based on these results, our findings demonstrate that niclosamide can impact multiple pathways involved in ALS. This multi-targeted approach leads to a slowdown in the progression of the disease, thereby positioning niclosamide as a promising candidate for repurposing in the treatment of ALS.
Published in
Neuroprotective effects of niclosamide on disease progression via inflammatory pathways modulation in SOD1-G93A and FUS-associated amyotrophic lateral sclerosis models
Milani M, Della Valle I, Rossi S et al. · Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics 2024 · PMID 38493058 · doi:10.1016/j.neurot.2024.e00346
This dataset
Open GSE249071 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 GSE249071_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 PRJNA1047112 and SRA study SRP475167. Searching any of these in the dataset finder brings you back here.

NCBI GEO page ↗ Paper (PMID 38493058) ↗
Study design
4 × spinal cord, veh end stage, vs 4 × spinal cord, nic end stage, vs 3 × spinal cord, veh symptomatic, vs 3 × spinal cord, nic symptomatic,

Supports a between-group comparison across 14 samples.

How we read this: 4 replicated groups read from 14 sample titles; they account for 14 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

Search all mouse RNA-seq datasets in GEO →

Share this dataset

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.