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Fibroblast growth factor receptor 1 (FGFR1) mRNA is the messenger RNA transcript that encodes the FGFR1 protein, a transmembrane receptor tyrosine kinase essential for cell growth, survival, and embryonic development [4, 8]. In various cancers, particularly squamous cell lung cancer and certain breast cancers, FGFR1 mRNA is frequently overexpressed, driving oncogenic signaling through the MAPK/ERK and PI3K/AKT pathways [1, 2, 4]. Unlike traditional small molecule inhibitors that target the FGFR1 protein's kinase domain, therapeutic strategies targeting the mRNA—such as antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs)—aim to silence the gene and prevent protein production entirely [12, 20, 22]. FGFR1 mRNA levels have also emerged as a superior predictive biomarker for identifying patients likely to respond to FGFR-targeted therapies compared to gene amplification alone [1, 2, 6]. Clinical development of mRNA-targeting agents like Ionis-FGFR1Rx has explored applications in both oncology and metabolic diseases, though challenges remain regarding tissue-specific delivery and managing systemic side effects like hyperphosphatemia [18, 20].
Antisense-mediated mRNA degradation (RNase H) [22], RNA interference (RNAi) [22]
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