AUH

Chr 9

AU RNA binding methylglutaconyl-CoA hydratase

This gene encodes bifunctional mitochondrial protein that has both RNA-binding and hydratase activities. The encoded protein is a methylglutaconyl-CoA hydratase that catalyzes the hydration of 3-methylglutaconyl-CoA to 3-hydroxy-3-methyl-glutaryl-CoA, a critical step in the leucine degradation pathway. This protein also binds AU-rich elements (AREs) found in the 3' UTRs of rapidly decaying mRNAs including c-fos, c-myc and granulocyte/ macrophage colony stimulating factor. ARE elements are involved in directing RNA to rapid degradation and deadenylation. This protein is localizes to the mitochondrial matrix and the inner mitochondrial membrane and may be involved in mitochondrial protein synthesis. Mutations in this gene are the cause of 3-methylglutaconic aciduria, type I. Alternative splicing results in multiple transcript variants. [provided by RefSeq, Sep 2015]

ResearchGenerating clinical summary…

Primary Disease Associations & Inheritance

UniProt3-methylglutaconic aciduria 1

Clinical highlights

Gene-disease validity (ClinGen)
3-methylglutaconic aciduria type 1 · ARDefinitivesufficient evidence for diagnostic panels
Interpreting a novel variant
Loss of function is the curated mechanism (Gene2Phenotype), though the gene is not strongly LoF-constrained in the population — weigh truncating variants against that tolerance.Curated gene-level mechanism — a prior for triage, not a per-variant call.
8
Active trials
26
Pubs (1 yr)
P/LP submissions
P/LP missense
0.96
LOEUF
LOF
Mechanism· G2P
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?
0.96LOEUF
pLI 0.000
Z-score 1.68
OE 0.58 (0.360.96)
Tolerant

Typical tolerance to LoF variation

Missense Constraint?
0.34Z-score
OE missense 0.93 (0.821.05)
169 obs / 182.0 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios?
LoF OE?0.58 (0.360.96)
00.351.4
Missense OE?0.93 (0.821.05)
00.61.4
Synonymous OE?0.97
01.21.6
LoF obs/exp: 11 / 18.9Missense obs/exp: 169 / 182.0Syn Z: 0.20

ClinVar Variant Classifications

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

AUH · 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

Safety and Impact of Medically Supervised Performance Enhancing Substance Usage in Healthy Elite Athletes

Impact of Medically Supervised Performance-Enhancing Substances (PES) on Elite Athletes

ENROLLING BY INVITATION
NCT07568574Phase NAEnhanced Emirates LimitedStarted 2026-03-12
Testosterone EnanthateTestosterone CypionateTestosterone Propionate
Male InfertilityMale Subfertility

Assessing the Correlation Between Phospholipase C Zeta Measurements and Semen Parameters

RECRUITING
NCT07684339Fakih IVF Fertility CenterStarted 2026-07-01
No Intervention: Observational Cohort
Philadelphia Chromosome-Positive Chronic Myeloid Leukemia

A Study to Investigate Tolerability and Efficacy of Asciminib (Oral) Versus Nilotinib (Oral) in Adult Participants (≥18 Years of Age) With Newly Diagnosed Philadelphia Chromosome Positive Chronic Myelogenous Leukemia in Chronic Phase (Ph+ CML-CP)

ACTIVE NOT RECRUITING
NCT05456191Phase PHASE3Novartis PharmaceuticalsStarted 2022-11-21
AsciminibNilotinib
Acute Myeloid Leukemia, Adult

Revumenib in Combination With Azacitidine + Venetoclax in Patients NPM1-mutated or KMT2A-rearranged AML

RECRUITING
NCT06652438Phase PHASE3Stichting Hemato-Oncologie voor Volwassenen NederlandStarted 2025-05-05
RevumenibPlacebo
Acute Myeloid Leukemia

Ivosidenib and Azacitidine With or Without Venetoclax in Adult Patients With Newly Diagnosed IDH1-Mutated AML or MDS/AML Considered Ineligible for Intensive Chemotherapy

RECRUITING
NCT07075016Phase PHASE3Stichting Hemato-Oncologie voor Volwassenen NederlandStarted 2025-08-05
Venetoclax 400Placebo
Hypercholesterolemia, Autosomal DominantHypercholesterolemia, Autosomal RecessiveFamilial Combined Hypercholesterolemia

Integrating Whole Genome Sequencing and Digital Twins Into the Management of Hypercholesterolemia in Emiratis

RECRUITING
NCT06535542Phase NAAbu Dhabi Health Services CompanyStarted 2024-07-29
Whole Genome Sequencing
HealthyHealthy Nutrition

The BEGIN Study Bifidobacterium Infantis to Newborns: Effects of Modulating the Gut Microbial Composition on Growth, Immune Function and Inflammatory Conditions - a Randomized Placebo-controlled Double-blinded Intervention Trial

RECRUITING
NCT06452199Phase NAUniversity of AarhusStarted 2024-06-10
B. infantisPlacebo
HematologyDiffused Large B Cell Lymphoma

A Multicenter Observational Study to Understand the Clinical Characteristics, Treatment Patterns and Access to Novel Therapies of Patients With Diffuse Large B-Cell Lymphoma in the MEA Region

RECRUITING
NCT07065344AstraZenecaStarted 2025-07-23