Target intelligence / Profile preview

Neurofilament medium polypeptide (NEFM)

Target
NEFM
Molecular classification
Intermediate filament protein, Cytoskeletal protein, Type IV intermediate filament
01

Overview

Neurofilament medium polypeptide (NEFM), also known as neurofilament medium chain or NF-M, is a principal component of the type IV intermediate filament heteropolymers specific to neurons[1][2][3][4]. NEFM is essential for maintaining the structure, size, and caliber of neuronal axons by forming the axoskeleton, alongside neurofilament light (NEFL) and heavy (NEFH) subunits[1][2][3][4]. NEFM regulates axonal growth and intracellular transport and plays important roles in the stability and function of synaptic connections, including interactions with dopamine D1 receptors and neurotransmitter signaling systems[3][4]. Aberrant expression, genetic variants, and post-translational modifications of NEFM are implicated in various neurodegenerative disorders, and its levels are widely used as a clinically validated biomarker for neuronal injury and neurodegeneration[1][2][3][4]. NEFM is not considered a direct therapeutic target but is a valuable tool in neurological disease diagnostics and research.

Other names
Neurofilament medium chainNEFMNEF3NFMNF-M160 kDa neurofilament proteinNeurofilament 3Neurofilament triplet M proteinneurofilament, medium polypeptide 150kDaneurofilament-3 (150 kD medium)
02

Mechanism of action

Not applicable; NEFM is not a direct target for current pharmacological therapies. Its modulation is not the primary therapeutic mechanism for existing drugs[4].

03

Biological functions

Maintenance of neuronal structure and caliberAxonal growth and stabilityIntracellular transport within neuronsRegulation of synaptic neurotransmissionModulation of dopaminergic signaling
04

Disease associations

Neurodegenerative disease (including amyotrophic lateral sclerosis, Charcot-Marie-Tooth disease, Alzheimer's disease, Parkinson's disease)NeuropathyPossible roles in some cancers (breast cancer, small cell osteogenic sarcoma)Neuropsychiatric disorders (e.g., schizophrenia)
05

Safety considerations

No direct safety concerns reported for targeting NEFM, as it is not a pharmacological target; primary challenge relates to reliability of biomarker detection in clinical populations[4].
06

Biomarkers

Biomarker of neuronal damageUsed in cerebrospinal fluid (CSF) and blood for diagnosis or monitoring of neurodegeneration (ALS, multiple sclerosis, Alzheimer's disease, etc.)[1][2][3][4]

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