GAN
Chr 16gigaxonin
Also known as: GAN1, GIG, KLHL16
This gene encodes a member of the cytoskeletal BTB/kelch (Broad-Complex, Tramtrack and Bric a brac) repeat family. The encoded protein plays a role in neurofilament architecture and is involved in mediating the ubiquitination and degradation of some proteins. Defects in this gene are a cause of giant axonal neuropathy (GAN). [provided by RefSeq, Oct 2008]
Primary Disease Associations & Inheritance
Clinical highlights
Some data sources returned errors (1)
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- scAAV9/JeT-GANAAV gene therapyPhase 1
Intrathecal delivery of the GAN gene.
Delivery: Intrathecal
Therapeutic landscape as of 2026-07. Educational only. Investigational ≠ available; not medical advice or eligibility. Approved entries are precise; investigational program names/phases are conservative and move fast. Curated from FDA/EMA approvals and the clinical-trial literature; verify against current labeling + ClinicalTrials.gov.
ClinicalTrials.govPopulation Genetics & Constraint
gnomAD v4 — loss-of-function & missense intolerance
More LoF-intolerant than ~75% of genes
Tolerant to missense variation
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.
External Resources
Links to major genomics databases and tools
Clinical Trials
Active and recruiting trials from ClinicalTrials.gov
A Study of Niraparib in Combination With Abiraterone Acetate and Prednisone Versus Abiraterone Acetate and Prednisone for the Treatment of Participants With Deleterious Germline or Somatic Homologous Recombination Repair (HRR) Gene-Mutated Metastatic Castration-Sensitive Prostate Cancer (mCSPC)
ACTIVE NOT RECRUITINGA Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1
RECRUITINGA Study to Evaluate the Efficacy and Safety of TL118 in Solid Tumors Patients
RECRUITINGDetermine Effectiveness of Anifrolumab In SYstemic Sclerosis (DAISY)
ACTIVE NOT RECRUITINGStudy of the Preventive Effects and Mechanisms of Yeast β-Glucan on Upper Respiratory Tract Infections
NOT YET RECRUITINGA Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)
ENROLLING BY INVITATIONPhase II Umbrella Study of Novel Anti-cancer Agents in Participants With NSCLC Who Progressed on an Anti-PD-1/PD-L1 Containing Therapy
ACTIVE NOT RECRUITINGEfficacy and Safety of Stapokibart in Non-Allergic Rhinitis With Eosinophilia Syndrome
NOT YET RECRUITINGRespiratory Muscles in End-stage Lung Disease: Pathophysiological Processes & Clinical Consequences
RECRUITINGNon-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis
RECRUITINGSingle Intravitreal (IVT) Injection of 4D-150 in Patients With Macular Neovascularization Secondary to Age-Related Macular Degeneration
RECRUITINGEfficacy and Safety of Olaparib (MK-7339) in Participants With Previously Treated, Homologous Recombination Repair Mutation (HRRm) or Homologous Recombination Deficiency (HRD) Positive Advanced Cancer (MK-7339-002 / LYNK-002)
ACTIVE NOT RECRUITINGExternal Resources
Links to major genomics databases and tools