Article
tRNA therapeutics for genetic diseases.
Nature reviews. Drug discovery - 1 Feb 2024
Coller Jeff, Ignatova Zoya
Abstract excerpt
Transfer RNAs (tRNAs) have a crucial role in protein synthesis, and in recent years, their therapeutic potential for the treatment of genetic diseases - primarily those associated with a mutation altering mRNA translation - has gained significant attention. Engineering tRNAs to readthrough nonsense mutation-associated premature termination of mRNA translation can restore protein synthesis and function. In...
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