GAN

Chr 16AR

gigaxonin

Also known as: GAN1, GIG, KLHL16

The protein functions as a cytoskeletal component that maintains neurofilament architecture and acts as a substrate-specific adapter for ubiquitin-mediated degradation of neuronal proteins including MAP1B and TBCB. Mutations cause giant axonal neuropathy-1, a progressive neurodegenerative disorder that typically presents in early childhood with peripheral neuropathy and distinctive giant axons containing neurofilament accumulations. This condition follows autosomal recessive inheritance.

OMIMResearchSummary from RefSeq, OMIM, UniProt
LOFmechanismARLOEUF 0.501 OMIM phenotype
Clinical SummaryGAN
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Gene-Disease Validity (ClinGen)
giant axonal neuropathy 1 · ARDefinitive

Definitive — sufficient evidence for diagnostic panels

Population Constraint (gnomAD)
Constrained for loss-of-function variants (OE-LoF 0.29) despite low pLI — interpret in context.
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Clinical Trials
12 active or recruiting trials — potential therapeutic options may be available

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Moderate LoF intolerance
LoF Constraint
0.50LOEUF
pLI 0.025
Z-score 3.71
OE 0.29 (0.170.50)
Moderately constrained

More LoF-intolerant than ~75% of genes

Missense Constraint
-0.73Z-score
OE missense 1.11 (1.021.21)
367 obs / 329.9 exp
Tolerant

Tolerant to missense variation

Observed / Expected Ratios
LoF OE0.29 (0.170.50)
00.351.4
Missense OE1.11 (1.021.21)
00.61.4
Synonymous OE1.55
01.21.6
LoF obs/exp: 9 / 31.4Missense obs/exp: 367 / 329.9Syn Z: -4.74
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
definitiveGAN-related giant axonal neuropathyLOFAR

Predictions shown for reference only — model trained on dominant genes, not applicable to AR conditions.

DN
0.6745th %ile
GOF
0.6834th %ile
LOF
0.2969th %ile

The Badonyi & Marsh prediction model was trained exclusively on dominant disease genes. Predictions are not reliable for genes with autosomal recessive inheritance and are shown at reduced opacity for reference only.

Predictions from Badonyi M, Marsh JA. PLoS ONE. 2024;19(8):e0307312.

ClinVar Variant Classifications

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

GAN · protein map & ClinVar variants

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

3D Protein StructureAlphaFold

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

Solid Tumor

A Study to Evaluate the Efficacy and Safety of TL118 in Solid Tumors Patients

RECRUITING
NCT06010342Phase PHASE2Teligene USStarted 2023-03-16
TL118 Capsule
Non-small Cell Lung Cancer

Study of Telisotuzumab Vedotin (ABBV-399) in Participants With Previously Treated c-Met+ Non-Small Cell Lung Cancer

ACTIVE NOT RECRUITING
NCT03539536Phase PHASE2AbbVieStarted 2018-10-10
Telisotuzumab vedotin
Esophageal Cancer

Investigating Multigene Methylation Dynamics in Treatment Response Surveillance for Esophageal Carcinoma

RECRUITING
NCT06979869Xijing HospitalStarted 2025-05-26
Multi-gene methylation testingMulti-gene methylation testingMulti-gene methylation testing
Non-small Cell Lung Cancer (NSCLC)

Sacituzumab Tirumotecan (MK-2870) Versus Chemotherapy in Previously Treated Advanced or Metastatic Nonsquamous Non-small Cell Lung Cancer (NSCLC) With EGFR Mutations or Other Genomic Alterations (MK-2870-004)

RECRUITING
NCT06074588Phase PHASE3Merck Sharp & Dohme LLCStarted 2023-11-12
Sacituzumab tirumotecanDocetaxelPemetrexed
Decompensated Cirrhosis

Human Umbilical Cord-derived Mesenchymal Stem Cells for Decompensated Cirrhosis (MSC-DLC-2)

RECRUITING
NCT05224960Phase PHASE2Beijing 302 HospitalStarted 2024-06-27
UC-MSCsPlacebo(solution without UC-MSCs)
Systemic SclerosisScleroderma

Determine Effectiveness of Anifrolumab In SYstemic Sclerosis (DAISY)

ACTIVE NOT RECRUITING
NCT05925803Phase PHASE3AstraZenecaStarted 2023-11-08
Anifrolumab (blinded)Placebo (blinded)Anifrolumab (unblinded, open label)
Leukemia, Myeloid, Acute

A Study of Bleximenib, Venetoclax and Azacitidine For Treatment of Participants With Newly Diagnosed Acute Myeloid Leukemia (AML)

RECRUITING
NCT06852222Phase PHASE3Janssen Research & Development, LLCStarted 2025-06-04
BleximenibVenetoclax (VEN)Azacitidine (AZA)
Neovascular Age-related Macular Degeneration

Study to Assess the Injection Burden, Adverse Events, Change in Disease Activity, and Long-Term Preservation of Visual Acuity of Surabgene Lomparvovec in Adult Participants With Neovascular Age-Related Macular Degeneration (nAMD)

RECRUITING
NCT07007065Phase PHASE3AbbVieStarted 2025-11-05
Surabgene Lomparvovec (ABBV-RGX-314)Ranibizumab Control
Locally Advanced Breast CancerMetastatic Breast Cancer

A Study to Evaluate the Effectiveness and Safety of Inavolisib in Participants With Endocrine-resistant, PIK3CA-mutated, Hormone Receptor-positive, HER2-negative Locally Advanced or Metastatic Breast Cancer

RECRUITING
NCT07347600Hoffmann-La RocheStarted 2026-01-21
InavolisibPalbociclibFulvestrant
Non-Small Cell Lung Cancer

A Clinical Trial of TQB2102 for Injection in Non-small Cell Lung Cancer With HER2 Gene Abnormality

RECRUITING
NCT06496490Phase PHASE2Chia Tai Tianqing Pharmaceutical Group Nanjing Shunxin Pharmaceutical Co., Ltd.Started 2024-08-06
TQB2102 for injectionTQB2102 for injection combined with Benmelstobart injection
Platinum SensitiveBRCA MutatedRelapsed Ovarian Cancer

Olaparib Treatment in BRCA Mutated Ovarian Cancer Patients After Complete or Partial Response to Platinum Chemotherapy

ACTIVE NOT RECRUITING
NCT01874353Phase PHASE3AstraZenecaStarted 2013-09-03
Olaparib 300mg tabletsPlacebo to match olaparib 300mg
Non-cystic Fibrosis Bronchiectasis

A Translational Study for Phenotyping and Endotyping Chinese Patients With NCFBE

RECRUITING
NCT07245407AstraZenecaStarted 2025-10-27
Participant Follow-up
Clinical Literature
Open Research Assistant →
Full-Text Mentions
NLP-detected gene mentions in article bodies · via PubTator3
PubTator3
BiVi-GAN: Bivariate Vibration GAN
Jeong H et al.·Sensors (Basel)
2024
Reciprocal GAN Through Characteristic Functions (RCF-GAN).
Li S et al.·IEEE Trans Pattern Anal Mach Intell
2022
GAN Compression: Efficient Architectures for Interactive Conditional GANs.
Li M et al.·IEEE Trans Pattern Anal Mach Intell
2022
Re-GAN: Data-Efficient GANs Training via Architectural Reconfiguration.
Saxena D et al.·IEEE Trans Pattern Anal Mach Intell
2025
Top 5 full-text resultsSearch PubTator3 ↗