RNA therapeutics target translation rather than DNA, aiming to correct shared protein production errors. By enabling cells to read through premature stop codons, engineered tRNAs could restore ...

Between 10 to 15% of all genetic diseases have the same underlying cause: a nonsense mutation. 1 This type of mutation results from a stop codon in the middle of a gene sequence, which terminates ...

Researchers develop a single genome-editing strategy to treat multiple disorders caused by nonsense mutations, promising efficient and cost-effective therapies.