GEO series

PROTACs therapeutically target the polyglutamine androgen receptor inspinal and bulbar muscular atrophy models

GSE297069 Homo sapiens (human) RNA-seq (gene expression) 24 samples Submitted 2025/12/09 Platform GPL24676

At a glance

RNA-seq (gene expression) · human · 24 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: 24 samples
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: PROTACs therapeutically target the polyglutamine androgen receptor in spinal and bulbar muscular atrophy modelsNeurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2025

Summary (written by the submitters)

Spinal and bulbar muscular atrophy (SBMA) is a CAG/polyglutamine (polyQ) repeat expansion disorder in which the mutant androgen receptor (AR) protein triggers progressive degeneration of the neuromuscular system in men. As the misfolded polyQ AR is the proximal mediator of toxicity, therapeutic efforts have focused on targeting the mutant protein, but these prior efforts have met with limited success in SBMA patients.

Read the full summary ↓

Here, we examine the efficacy of proteolysis targeting chimeras (PROTACs), small molecule AR degraders that rapidly and potently promote AR ubiquitination and degradation by the proteasome. We identify ARD-1676 as a PROTAC that clears polyQ AR in an over-expression system, in patient iPSC-derived induced motor neurons and skeletal muscle cells, and in a gene targeted mouse model of disease. Furthermore, we demonstrate that 24-hour treatment with ARD-1676 rescues transcriptional dysregulation in SBMA induced skeletal muscle cells. These data provide evidence of therapeutic efficacy and in vivo target engagement, establishing PROTACs as potential therapeutic agents for the treatment of SBMA.

Published in

PROTACs therapeutically target the polyglutamine androgen receptor in spinal and bulbar muscular atrophy models

Sangotra A, Reddy SL, Kuo CJ et al. · Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics 2025 · PMID 40912964 · doi:10.1016/j.neurot.2025.e00732

This dataset

Open GSE297069 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 GSE297069_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 PRJNA1262438 and SRA study SRP584918. Searching any of these in the dataset finder brings you back here.

NCBI GEO page ↗ Paper (PMID 40912964) ↗

Samples in this study

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+ 24 more — browse all 24 samples with per-sample file links →

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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.