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RNA-seq studies in dabrafenib treated endothelial cells

GSE329903 Homo sapiens (human) RNA-seq (gene expression) 12 samples Submitted 2026/07/15 Platform GPL24676
At a glance

RNA-seq (gene expression) · human · 12 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
12 samples: 3 × HUVEC shCCM3 dabrafenib treated 48h n vs 3 × HUVEC shCCM3 vehicle treated 48h n vs 3 × HUVEC shScramble dabrafenib treated 48h n vs 3 × HUVEC shScramble vehicle treated 48h nGroups 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)
Cerebral Cavernous Malformations (CCM) constitute the second most common type of intracranial vascular malformations. Cavernomas arise due to somatic or autosomal dominant inherited loss-of-function mutations in KRIT1 (CCM1), CCM2, or PDCD10 (CCM3), forming fragile mulberry-like malformed blood vessel structures, which are prone to leak and break.
Read the full summary ↓
Treatment is limited to surgical removal of solitary cavernomas. As patients often present with multiple lesions, there is an urgent need to develop non-invasive therapeutic strategies. We performed a high throughput screen of 5200 bioactive molecules followed by image analysis on Ccm3-knockout endothelial cells and discovered that the FDA-EMA-approved BRAF inhibitor dabrafenib, effectively restored cell-cell junctions and vascular barrier properties of CCM-deficient endothelial cells. The potential translatability of dabrafenib was tested in vivo, where it reduced cerebral lesion burden and suppressed lesion permeability, reducing perivascular plasma protein depositions. Dabrafenib also rescued abnormal structural defects present in the developing retina vasculature. Mechanistically, dabrafenib targeted extracellular regulated kinase (ERK)5-Krüppel like factor (KLF)2/4 signaling pathway and suppressed F-actin stress fiber formation, two potentially independent and central molecular processes involved in CCM pathology. Dabrafenib is a kinase inhibitor indicated for the treatment of tumors with BRAF V600E mutations. However, silencing of BRAF did not interfere with dabrafenib-induced KLF2/4 inhibition and restoration of cell-cell junctions, suggesting that its dual therapeutic outcome is independent of BRAF pathway inhibition. In conclusion, dabrafenib is a small molecular weight compound with marked inhibitory effects on cavernoma pathogenesis suggesting that CCMs could be treated with dabrafenib to block cavernoma growth and vascular leakage.
This dataset
Open GSE329903 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 GSE329903_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 PRJNA1462056 and SRA study SRP697327. Searching any of these in the dataset finder brings you back here.

NCBI GEO page ↗
Study design
3 × HUVEC shCCM3 dabrafenib treated 48h n vs 3 × HUVEC shCCM3 vehicle treated 48h n vs 3 × HUVEC shScramble dabrafenib treated 48h n vs 3 × HUVEC shScramble vehicle treated 48h n

Supports a between-group comparison across 12 samples.

How we read this: 4 replicated groups read from 12 sample titles; they account for 12 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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