{"database":"GEO","file_versions":[{"headers":{"Content-Type":["application/json"]},"body":{"files":{"Other":["ftp://ftp.ncbi.nlm.nih.gov/geo/series/GSE289nnn/GSE289932/"]},"type":"primary"},"statusCode":"OK","statusCodeValue":200}],"scores":null,"additional":{"omics_type":["Transcriptomics"],"species":["Homo sapiens"],"gds_type":["Expression profiling by high throughput sequencing"],"full_dataset_link":["https://www.ncbi.nlm.nih.gov/geo/query/acc.cgi?acc=GSE289932"],"repository":["GEO"],"entry_type":["GSE"],"additional_accession":[]},"is_claimable":false,"name":"Anti-inflammatory and phosphorylation effects of CFTR modulator triple therapy in cystic fibrosis","description":"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. 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. 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.","dates":{"publication":"2026/08/03"},"accession":"GSE289932","cross_references":{"GSM":["GSM8801794","GSM8801795","GSM8801796","GSM8801830","GSM8801797","GSM8801831","GSM8801798","GSM8801810","GSM8801799","GSM8801832","GSM8801811","GSM8801833","GSM8801834","GSM8801812","GSM8801813","GSM8801814","GSM8801815","GSM8801816","GSM8801817","GSM8801818","GSM8801819","GSM8801791","GSM8801792","GSM8801793","GSM8801820","GSM8801821","GSM8801822","GSM8801800","GSM8801823","GSM8801801","GSM8801802","GSM8801824","GSM8801825","GSM8801803","GSM8801826","GSM8801804","GSM8801805","GSM8801827","GSM8801828","GSM8801806","GSM8801807","GSM8801829","GSM8801808","GSM8801809"],"GPL":["24676"],"GSE":["289932"],"taxon":["Homo sapiens"],"PMID":["[42707060]"]}}