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Fibroblast growth factor receptor 3 (FGFR3) messenger RNA is the transcript responsible for the synthesis of the FGFR3 protein, a key regulator of bone growth and cellular signaling [NCBI Gene, 2024]. Mutations in the FGFR3 gene, particularly gain-of-function mutations like G380R, lead to overactive signaling that inhibits chondrocyte proliferation, resulting in skeletal dysplasias such as achondroplasia [StatPearls, 2023]. In oncology, FGFR3 mRNA overexpression or specific fusions are implicated in the pathogenesis of bladder cancer and multiple myeloma [PubMed, 2021]. Targeting the mRNA directly using antisense oligonucleotides (ASOs) or small interfering RNAs (siRNAs) offers a strategy to downregulate the production of the mutant or overexpressed protein [PubMed, 2017]. This approach aims to restore normal bone growth in genetic disorders or inhibit tumor progression in malignancies. Current research focuses on improving the delivery and stability of these RNA-targeted therapies to ensure therapeutic efficacy while minimizing off-target effects [Nature Reviews Drug Discovery, 2022].
Antisense inhibition of translation, RNA interference-mediated degradation, or splice modulation to produce non-functional or truncated protein isoforms [Nature Reviews Drug Discovery, 2022].
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