Target intelligence / Profile preview

Fibroblast growth factor 14 (FGF14)

Target
FGF14
Molecular classification
Growth factor (non-secreted/atypical FGF family member), Intracellular signaling molecule, Ion channel regulatory protein (regulator of voltage-gated sodium channels), Other (not a classical receptor, enzyme, or transporter)
01

Overview

Fibroblast growth factor 14 (FGF14) is an atypical member of the fibroblast growth factor family, functioning as an intracellular protein primarily expressed in the central nervous system[1][2][3][4]. Unlike canonical FGFs, FGF14 does not act through cell surface FGF receptors but instead interacts directly with the intracellular domains of voltage-gated sodium (Na\(_v\)) channels, regulating their gating, localization, and neuronal excitability[1]. It is required for synaptic transmission, synaptic plasticity, and neurogenesis, and plays a fundamental role in neuronal and cognitive functions[1][2][3]. Mutations or deletions in the FGF14 gene are associated with spinocerebellar ataxia type 27, episodic ataxia, and have been implicated in a spectrum of cognitive, affective, and motor disorders, including schizophrenia, depression, and Alzheimer’s disease[1][2][3][4]. FGF14 is sometimes referred to as fibroblast growth factor homologous factor 4 (FHF4), SCA27, or NYS4, reflecting its disease linkages and genetic locus. Currently, no drugs are clinically approved to target FGF14 directly, but its role as a regulator of ion channel function and neuronal signaling makes it a subject of ongoing research in neurobiology and neurotherapeutics[1][2][4].

Other names
FHF4FGF-14FHF-4NYS4SCA27SCA27ASCA27Bfibroblast growth factor homologous factor 4nystagmus 4 congenital autosomal dominant
02

Biological functions

Regulation of neuronal excitability (via modulation of voltage-gated Na+ channels)Synaptic transmissionSynaptic plasticityNeurogenesis (maturation of hippocampal progenitor cells)Nervous system development and function
03

Disease associations

Neurodegenerative disease (e.g., Spinocerebellar ataxia type 27, SCA27/27A/27B)SchizophreniaDepressionAnxietyAddictive behaviorsAlzheimer's disease (potential biomarker / disease association)Other neurological and psychiatric disorders
04

Safety considerations

Genetic variants can cause or predispose to neurodegenerative and neurodevelopmental disorders (e.g., ataxia, nystagmus, cognitive deficits)No specific drug safety concerns described, as no direct clinical drugs target FGF14 yet
05

Biomarkers

FGF14 expression (potential biomarker in Alzheimer's disease and possibly other neuropsychiatric conditions)

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