For disorders driven by a known genetic component, research understandably focuses on developing a drug targeting that genetic signature. When that works, the outcome benefits patients but the end ...

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 ...

A new advanced, high-throughput sequencing technique should enable scientists to have a firmer grasp on the activity of naturally occurring microbiomes in response to real-world conditions and diet.

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