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Target intelligence / Profile preview
Mitochondrial tRNA-specific 2-thiouridylase 1 (TRMU)
- Target
- TRMU
- Molecular classification
- Enzyme, tRNA-modifying enzyme, Thiouridylase
01
Overview
Mitochondrial tRNA-specific 2-thiouridylase 1 (TRMU) is a nuclear-encoded mitochondrial enzyme that catalyzes the post-transcriptional 2-thiolation of the wobble uridine (U34) in mitochondrial tRNAs for lysine, glutamate, and glutamine. This modification is essential for the accuracy and efficiency of mitochondrial translation, particularly the synthesis of 13 respiratory chain subunits from mitochondrial DNA. Genetic defects in TRMU impair this modification and are causally linked to acute infantile liver failure, certain syndromic mitochondrial encephalopathies, and complications in other metabolic disorders. While no drugs currently target TRMU directly, its activity and genetic mutations serve as important biomarkers for patient selection and disease characterization in mitochondrial medicine
- Other names
- MTU1MTO2 homologTRMU protein, humantRNA 5-methylaminomethyl-2-thiouridylate methyltransferaseTRMTTRMT1TRNT1LCAL3
02
Mechanism of action
Not applicable; drugs targeting this enzyme have not been described in the literature. Hypothetically, small molecule inhibitors or gene therapies could modulate TRMU, but no such agents are currently reported
03
Biological functions
Catalyzes the 2-thiolation of uridine at the wobble position (U34) of mitochondrial tRNA(Lys), tRNA(Glu), and tRNA(Gln)Post-transcriptional modification of mitochondrial tRNAsRegulation of mitochondrial protein synthesis
04
Disease associations
Acute infantile liver failure (associated with mutations)Myoclonic epilepsy with ragged-red fibers (MERRF)Mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episode (MELAS) syndromeOther mitochondrial diseases related to defects in tRNA modification
05
Safety considerations
Loss-of-function mutations can cause acute liver failure in infants, especially under metabolic stress or transient cysteine deficiencyPotential off-target/misregulation risks in gene therapy approaches due to its central role in mitochondrial translation
06
Biomarkers
Reduced 2-thiolation levels of mitochondrial tRNAs (Lys, Glu, Gln) as a functional readoutGenetic mutations in TRMU detectable by sequencing for inherited mitochondrial disorders
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