Omics · study · 2026
Anti-inflammatory and phosphorylation effects of CFTR modulator triple therapy in cystic fibrosis
Listed in NCBI GEO
Cystic Fibrosis is an autosomal recessive disorder caused by mutations in the CFTR gene, which encodes a key chloride channel, leading to impaired ion transport and severe progressive respiratory failure.
Description
CFTR modulators, such as Trikafta© has improved morbidity in CF, but its systemic effects remain unclear. This study evaluated the clinical outcomes, safety, and cellular mechanisms of Trikafta© in young children (< 11 years) with CF using a multi-omic approach.
Seventeen children with CF (median age 8.7 years, 26% female) and 12 healthy controls (median age 9.6 years, 42% female) were included for multi-omic analysis. The analysis included RNA sequencing, proteomics and phosphoproteomics. Baseline pathway enrichment analysis (FDR < 0.05) revealed systemic inflammation, transmembrane transporter deficiencies, reduced protein kinase, and reduced GTPase activity.
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Three months post-Trikafta© treatment, anti-inflammatory effects, epigenetic modulation, and increased protein kinase activity were observed, indicating partial reversal of cellular abnormalities. Trikafta© improves clinical outcomes and reduces inflammation, epigenetic changes and key phosphorylation pathways in young children with CF, offering insights into CF pathology and potential biomarkers for monitoring treatment.
Links
Get the data
- GEO FTP directory ftp.ncbi.nlm.nih.gov/geo/series/GSE289nnn/GSE289932 ↗
download · from NCBI GEO
Where it is published
- GEO accession page ncbi.nlm.nih.gov/geo/query/acc.cgi?acc=GSE289932 ↗
landing page · from NCBI GEO
Documentation and papers
- PRJNA1225598 ncbi.nlm.nih.gov/bioproject/PRJNA1225598 ↗
project · from NCBI GEO
- PubMed 42707060 pubmed.ncbi.nlm.nih.gov/42707060 ↗
publication · from NCBI GEO
Topics
- Stated by source
- Expression profiling by high throughput sequencing · Homo sapiens
- From keywords
- Life Sciences
- Inferred from text
- Mass spectrometry 65% · RNA sequencing 75% · Sequencing 75%
Provenance · 1 source records, 10 field assertions
| Source | Key | Last seen | Raw |
|---|---|---|---|
| NCBI GEO | GSE289932 | 15 d ago | JSON v1 |
| Field | Assertion | Extractor | Evidence |
|---|---|---|---|
| access_level | source · NCBI GEO | connector:ncbi_geo@1.0.0 | |
| concepts[field].local:field:life-sciences | mapping · NCBI GEO | connector:ncbi_geo@1.0.0 | |
| concepts[method].geo_series_type:expression-profiling-by-high-throughput-sequencing | source · NCBI GEO | connector:ncbi_geo@1.0.0 | /gdstype |
| concepts[modality].local:modality:mass-spectrometry | enrichment · NCBI GEO | keyword-concept-rules@1.0.0 | title+description (65%) |
| concepts[modality].local:modality:rna-seq | enrichment · NCBI GEO | keyword-concept-rules@1.0.0 | title+description (75%) |
| concepts[modality].local:modality:sequencing | enrichment · NCBI GEO | keyword-concept-rules@1.0.0 | title+description (75%) |
| concepts[organism].NCBITaxon:9606 | source · NCBI GEO | connector:ncbi_geo@1.0.0 | /taxon |
| description | source · NCBI GEO | connector:ncbi_geo@1.0.0 | /summary |
| publication_date | source · NCBI GEO | connector:ncbi_geo@1.0.0 | |
| title | source · NCBI GEO | connector:ncbi_geo@1.0.0 | /title |