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Target intelligence / Profile preview
Premature Termination Codon (Arg-TGA) (Arg-TGA PTC)
- Target
- Arg-TGA PTC
- Molecular classification
- Genetic mutation, Stop codon, mRNA
01
Overview
A premature termination codon (PTC), specifically the Arg-TGA mutation, refers to a genetic alteration where an arginine codon (CGA or AGA) is mutated to a TGA stop codon. This leads to premature cessation of translation and production of truncated, typically nonfunctional proteins. It is implicated in numerous genetic diseases and is a target for readthrough therapies.
- Other names
- Arginine to TGA nonsense mutationCGA/AGA to TGA mutationTGA stop codon mutationPremature stop codon
02
Mechanism of action
Engineered tRNAs enable translational readthrough at the PTC site and restore arginine incorporation into nascent polypeptides.
03
Biological functions
Termination of translationProtein synthesis disruptionmRNA degradation (NMD)Gene expression regulation
04
Disease associations
Genetic disordersStop codon diseasesInherited metabolic disorders
05
Safety considerations
Off-target effects of readthrough therapiesImmunogenicity of engineered tRNAsPotential for unintended protein modifications
06
Interacting drugs
AP003
1 more in the full profile.
07
Biomarkers
Presence of Arg-TGA mutation
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