Science

Unlike traditional approaches, this new technology does not require delivery of exogenous editing enzymes. Using only a segment of engineered RNA molecules, it can mobilize key enzymes that naturally exist in human cells to achieve precise editing of target RNA. The system is simple with high safety, imposing minimal delivery burden.

The team has collaborated with Kunming University of Science and Technology in Southwest China, Shanghai Jiao Tong University in East China and other institutions to develop candidate drugs. Three pediatric patients who had received treatment all demonstrated significant and sustained improvements in motor function during the one-year follow-up period.