Chinese researchers treat rare disease via new RNA editing technology
Chinese researchers from the Changping Laboratory and Peking University developed LEAPER, an RNA editing technology that successfully treated Duchenne muscular dystrophy (DMD) in clinical trials, marking the first global application of RNA editing for this disease. Three pediatric patients showed sustained motor function improvements after one-year follow-ups, with the technology offering a safer, enzyme-free approach compared to traditional gene therapy methods.
A Chinese research team has made a breakthrough in treating Duchenne muscular dystrophy (DMD) using a new RNA editing technology called LEAPER. Developed by scientists from the Changping Laboratory and Peking University, this technology entered clinical trials in China, becoming the first RNA editing method globally applied to DMD treatment. DMD, a severe hereditary disease caused by gene mutations, leads to progressive muscle atrophy and loss of motor function. Current treatments only delay progression without reversing the disease, and its large causative gene makes conventional gene therapy ineffective. The LEAPER system precisely locates and corrects genetic errors in muscle cells, restoring functional protein production without requiring external editing enzymes. The technology uses engineered RNA molecules to activate naturally occurring human enzymes, ensuring high safety and minimal delivery burden. Collaborating with Kunming University of Science and Technology and Shanghai Jiao Tong University, the team tested candidate drugs on three pediatric patients, all showing significant and sustained motor function improvements during a one-year follow-up. Team leader Wei Wensheng explained that LEAPER restores the genetic blueprint in muscle cells, helping muscles regain vitality. Director Xie Xiaoliang emphasized the platform’s potential to benefit patients with major diseases through original research and cutting-edge technology. The breakthrough reflects China’s advancements in gene therapy and RNA editing, offering hope for broader applications in treating genetic disorders.
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