Target intelligence / Profile preview

Fibroblast growth factor receptor 3 messenger RNA (FGFR3 mRNA) (FGFR3 mRNA)

Target
FGFR3 mRNA
Molecular classification
Nucleic acid, Messenger RNA
01

Overview

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].

Other names
FGFR3 transcriptCD333 mRNAACH mRNACEK2 mRNAJTK4 mRNAHSFGFR33 mRNA
02

Mechanism of action

Antisense inhibition of translation, RNA interference-mediated degradation, or splice modulation to produce non-functional or truncated protein isoforms [Nature Reviews Drug Discovery, 2022].

03

Biological functions

Protein synthesisRegulation of chondrocyte proliferationBone developmentSignal transduction
04

Disease associations

AchondroplasiaBladder cancerMultiple myelomaThanatophoric dysplasiaHypochondroplasiaMuenke syndrome
05

Safety considerations

Off-target hybridizationDelivery to chondrocytes or tumor cellsSystemic toxicity of oligonucleotide chemistryPotential for over-suppression of normal bone growth
06

Interacting drugs

Antisense oligonucleotides (experimental)

2 more in the full profile.

07

Biomarkers

FGFR3 G380R mutationFGFR3 mRNA expression levelsFGFR3-TACC3 fusion transcript

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