GNE

Chr 9

glucosamine (UDP-N-acetyl)-2-epimerase/N-acetylmannosamine kinase

Also known as: DMRV, GLCNE, IBM2, NM, THC12, Uae1

The protein encoded by this gene is a bifunctional enzyme that initiates and regulates the biosynthesis of N-acetylneuraminic acid (NeuAc), a precursor of sialic acids. It is a rate-limiting enzyme in the sialic acid biosynthetic pathway. Sialic acid modification of cell surface molecules is crucial for their function in many biologic processes, including cell adhesion and signal transduction. Differential sialylation of cell surface molecules is also implicated in the tumorigenicity and metastatic behavior of malignant cells. Mutations in this gene are associated with sialuria, autosomal recessive inclusion body myopathy, and Nonaka myopathy. Alternative splicing of this gene results in transcript variants encoding different isoforms. [provided by RefSeq, Jul 2008]

GeneReviewsResearchGenerating clinical summary…

Primary Disease Associations & Inheritance

UniProtSialuria
UniProtNonaka myopathy
UniProtThrombocytopenia 12 with or without myopathy

Clinical highlights

Gene-disease validity (ClinGen)
macrothrombocytopenia, isolated · ARDefinitivesufficient evidence for diagnostic panels
12
Active trials
122
Pubs (1 yr)
P/LP submissions
P/LP missense
1.16
LOEUF
Mechanism
📖
GeneReview available — GNE
Authoritative clinical overview · Recommended first read
Open GeneReview ↗
Some data sources returned errors (1)

omim: Error: OMIM fetch failed: 429

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint?
1.16LOEUF
pLI 0.000
Z-score 0.83
OE 0.84 (0.621.16)
Tolerant

Highly tolerant — LoF variants common in population

Missense Constraint?
2.59Z-score
OE missense 0.64 (0.580.71)
271 obs / 420.5 exp
Mild constraint

Moderately missense-constrained (top ~2.5%)

Observed / Expected Ratios?
LoF OE?0.84 (0.621.16)
00.351.4
Missense OE?0.64 (0.580.71)
00.61.4
Synonymous OE?0.84
01.21.6
LoF obs/exp: 27 / 32.1Missense obs/exp: 271 / 420.5Syn Z: 1.59

ClinVar Variant Classifications

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

GNE · 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

Locally Advanced or Metastatic Conventional Chondrosarcoma With an IDH1 Mutation, Untreated or Previously Treated With 1 Systemic Treatment Regimen

Ivosidenib in Participants With Locally Advanced or Metastatic Conventional Chondrosarcoma Untreated or Previously Treated With 1 Systemic Treatment Regimen

RECRUITING
NCT06127407Phase PHASE3Servier Bio-Innovation LLCStarted 2024-07-09
Ivosidenib 500mgPlacebo
Type 1 Interferonopathies

A Study to Test How Well BI 3000202 is Tolerated by People With Type 1 Interferonopathies

ACTIVE NOT RECRUITING
NCT06878365Phase PHASE1Boehringer IngelheimStarted 2025-07-29
BI 3000202_low doseBI 3000202_high dose
X-Linked Retinitis Pigmentosa

Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

ACTIVE NOT RECRUITING
NCT04794101Phase PHASE3Janssen Research & Development, LLCStarted 2020-12-04
Genetic: AAV5-hRKp.RPGR Intermediate DoseGenetic: AAV5-hRKp.RPGR Low Dose
VitiligoNon-Segmental Vitiligo (NSV)

A Study of LY4005130 in Adult Participants With Non-Segmental Vitiligo

RECRUITING
NCT07533019Phase PHASE2Eli Lilly and CompanyStarted 2026-04-14
LY4005130Placebo
Pancreatic Cancer

Prediction of Surgical Resectability After FOLFIRINOX Chemotherapy for Borderline Resectable and Locally Advanced Pancreatic Cancer: the Role of Diffusion Weighted Magnetic Resonance Imaging, Radiomics and Liquid Biopsy (PeRFormanCe Trial)

RECRUITING
NCT05298722Phase NAUniversity Hospital, GhentStarted 2022-12-12
Imaging analysis of CT-scan and MRI with radiomics and genetic analysis of peripheral blood samples and questionnaires.
Cystic Fibrosis

Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del

ACTIVE NOT RECRUITING
NCT05331183Phase PHASE3Vertex Pharmaceuticals IncorporatedStarted 2022-11-23
ELX/TEZ/IVAIVA
Breast NeoplasmsTriple Negative Breast NeoplasmsHR Low-Positive/HER2-Negative Breast Neoplasms

A Clinical Study of Sacituzumab Tirumotecan (Sac-TMT, MK-2870) in People With Breast Cancer (MK-2870-032)

RECRUITING
NCT06966700Phase PHASE3Merck Sharp & Dohme LLCStarted 2025-06-30
Sacituzumab tirumotecanPembrolizumabRescue Medication
Prostate Cancer

A Study of Metastases Free Survival With Saruparib vs Placebo Added to a Standard RT/ADT in Men With High-risk Prostate Cancer With a BRCA Mutation

RECRUITING
NCT06952803Phase PHASE3AstraZenecaStarted 2025-08-06
SaruparibPlaceboAbiraterone + Prednisolone/Prednisone
X-Linked Hypophosphatemia

Registry for Patients With X-Linked Hypophosphatemia

RECRUITING
NCT03193476Kyowa Kirin Pharmaceutical Development LtdStarted 2017-09-12
Retinitis Pigmentosa (RP)Rod Cone Dystrophy

Open-Label Study to Evaluate the Safety, Tolerability, and Efficacy of Gene Therapy (SPVN20) in Subjects With Rod-Cone Dystrophy

RECRUITING
NCT07807111Phase PHASE1, PHASE2SparingVisionStarted 2025-10-08
SPVN20 (high dose)SPVN20 (medium dose)SPVN20 (low dose)
Retinitis Pigmentosa (RP)Usher Syndrome Type 2Deaf Blind

Study to Evaluate Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene

ACTIVE NOT RECRUITING
NCT06627179Phase PHASE2Laboratoires TheaStarted 2024-12-11
Intravitreal Injection of UltevursenNo intervention, will not receive any active study intervention
Cytomegalovirus (CMV)

A Study of Maribavir in Adults With Post-transplant Cytomegalovirus (CMV) Infection in Belgium

ACTIVE NOT RECRUITING
NCT06677892TakedaStarted 2025-02-24
No Intervention