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

Abstract

A human tRNALys gene was converted to an amber suppressor by site-specific mutagenesis of the anticodon. The mutated tRNALys gene directed synthesis of a tRNA that suppressed the UAG amber nonsense mutation in β0 thalassaemia mRNA. Such genes may be used to detect other nonsense mutations in mammalian cells and may provide an approach to gene therapy for β0 thalassaemia due to nonsense mutations.

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Temple, G., Dozy, A., Roy, K. et al. Construction of a functional human suppressor tRNA gene: an approach to gene therapy for β-thalassaemia. Nature 296, 537–540 (1982). https://doi.org/10.1038/296537a0

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