Construction of a functional human suppressor tRNA gene: an approach to gene therapy for β-thalassaemia

A human tRNA Lys gene was converted to an amber suppressor by site-specific mutagenesis of the anticodon. The mutated tRNA Lys gene directed synthesis of a tRNA that suppressed the UAG amber nonsense mutation in β 0 thalassaemia mRNA. Such genes may be used to detect other nonsense mutations in mammalian cells and may provide an approach to gene therapy for β 0 thalassaemia due to nonsense mutations.