NEFL

Chr 8ADAR

neurofilament light chain

Also known as: CMT1F, CMT2E, CMTDIG, NF-L, NF68, NFL, PPP1R110

Neurofilaments are type IV intermediate filament heteropolymers composed of light, medium, and heavy chains. Neurofilaments comprise the axoskeleton and they functionally maintain the neuronal caliber. They may also play a role in intracellular transport to axons and dendrites. This gene encodes the light chain neurofilament protein. Mutations in this gene cause Charcot-Marie-Tooth disease types 1F (CMT1F) and 2E (CMT2E), disorders of the peripheral nervous system that are characterized by distinct neuropathies. A pseudogene has been identified on chromosome Y. [provided by RefSeq, Oct 2008]

GeneReviewsOMIMResearchGenerating clinical summary…

Primary Disease Associations & Inheritance

Charcot-Marie-Tooth disease, dominant intermediate GMIM #617882
AD
Charcot-Marie-Tooth disease, type 1FMIM #607734
ADAR
Charcot-Marie-Tooth disease, type 2EMIM #607684
AD

Clinical highlights

Gene-disease validity (ClinGen)
Charcot-Marie-Tooth disease type 2 · ARDefinitivesufficient evidence for diagnostic panels2 gene-disease associations curated in total
12
Active trials
86
Pubs (1 yr)
P/LP submissions
P/LP missense
LOEUF
Multiple*
Mechanism· predicted
📖
GeneReview available — NEFL
Authoritative clinical overview · Recommended first read
Open GeneReview ↗

Population Genetics & Constraint

Constraint data not available from gnomAD.

ClinVar Variant Classifications

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

NEFL · protein map & ClinVar variants

Showing all ClinVar variants across the protein. Search a specific variant to highlight its position.

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

Alzheimer Disease (AD)

Tributyrin Treatment in Mild Alzheimer Disease: Assessment of Butyrate Effects Via the Gut-Brain

NOT YET RECRUITING
NCT06797817Phase PHASE3Universidad de AlmeriaStarted 2026-06-01
tributyrinPlacebo
Hereditary Ataxia

Hereditary Ataxia Research on Multi-Omics and Neuroclinical Insights in the Yangtze Delta

RECRUITING
NCT07092358Second Affiliated Hospital, Zhejiang University, School of MedicineStarted 2025-06-01
None of intervention
Lupus

Exploring Biological Mechanisms Underlying the TEACH Intervention for Adolescents With Lupus

NOT YET RECRUITING
NCT07771088Phase NAThe Hospital for Sick ChildrenStarted 2026-09-01
TEACH
Huntington Disease

A Study to Investigate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease

RECRUITING
NCT07326709Phase PHASE3Novartis PharmaceuticalsStarted 2026-03-24
Votoplam (blinded)Placebo
Amyotrophic Lateral Sclerosis (ALS)Mutation in the Superoxide Dismutase-1 (SOD1) Gene

First in Human (FIH) Study of ALN-SOD in Adult Participants With Amyotrophic Lateral Sclerosis Associated With Mutation in the SOD1 Gene (SOD1-ALS)

RECRUITING
NCT06351592Phase PHASE1, PHASE2Regeneron PharmaceuticalsStarted 2024-08-28
ALN-SODDiluentPlacebo (PB)
Transthyretin AmyloidosisATTR-CMATTRv-PN

Non-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis

RECRUITING
NCT06465810AstraZenecaStarted 2024-06-25
Treatment of transthyretin (ATTR) amyloidosis in observational study setting
Amyotrophic Lateral Sclerosis

Personalized Antisense Oligonucleotide Therapy for A Single Patient With CHCHD10 ALS (nL18576)

NOT YET RECRUITING
NCT07423494Phase PHASE1, PHASE2n-Lorem FoundationStarted 2026-03
nL-CHCHD-001
Mild Cognitive ImpairmentAlzheimer Disease

Cognitive Vulnerability to Stress in Individuals at Risk for Alzheimer's Disease

RECRUITING
NCT05795634Phase NAJohns Hopkins UniversityStarted 2023-03-01
Trier Social Stress Test
Huntington Disease

A Study to Evaluate AB-1001 Striatal Administration in Adults With Early Manifest Huntington's Disease

ACTIVE NOT RECRUITING
NCT05541627Phase PHASE1, PHASE2AskBio France, SAS, a subsidiary of AskBio Inc.Started 2022-10-12
AB-1001 Gene Therapy
Multiple Sclerosis

Understanding the Impact of Meal Timing on Neurological Health in Adults With Multiple Sclerosis

NOT YET RECRUITING
NCT07475377Phase NAUniversity of Alabama at BirminghamStarted 2026-08-01
Time Restricted EatingUnrestricted eating
ALS (Amyotrophic Lateral Sclerosis)

Safety and Tolerability Study of CTx1000 In Participants With Amyotrophic Lateral Sclerosis

RECRUITING
NCT07401121Phase PHASE1Celosia Therapeutics Pty LtdStarted 2026-01-12
AAV9 Gene therapy
Multiple Sclerosis

This is an Early Exploratory Study to Assess the Tolerability and Safety of GC012F in Patients With Multiple Sclerosis

RECRUITING
NCT07303790Phase EARLY_PHASE1Daishi TianStarted 2026-03-09
GC012F CAR-T Cell Injection