BEIJING, June 10 (Xinhua) -- A Chinese research team has achieved a breakthrough with its newly developed RNA editing technology, named LEAPER, in the treatment of Duchenne muscular dystrophy (DMD), a severe and rare hereditary disease. Both details of this technology and its clinical application were published on Wednesday in the journal Cell.
This marks the first time China-developed RNA editing technology has entered clinical trials, while it is also the first time globally that RNA editing has been applied in DMD treatment.
DMD is caused by gene mutations, with symptoms including progressive muscle atrophy and loss of motor function. Patients typically develop symptoms in childhood, gradually lose the ability to walk, and most die prematurely from respiratory or heart failure. Although classified as a rare disease, China has one of the world's largest DMD patient populations due to its vast population base. Current treatments can only partially delay disease progression but cannot reverse the course of the illness.
DMD is also widely recognized as one of the most challenging diseases in the field of gene therapy. Its causative gene is too large for conventional gene therapy methods to deliver the complete gene into patients' bodies.
In addition, different patients carry more than 7,000 types of pathogenic mutations, meaning a single treatment regimen can often only cover a small fraction of patients. Therefore, developing treatment strategies that combine broad applicability with long-term efficacy has remained a major challenge for the global medical community.
The new RNA editing technology was developed by a team from the Beijing-based Changping Laboratory and Peking University. According to team leader Wei Wensheng, LEAPER system components can precisely locate the erroneous genetic "blueprint" in muscle cells, restore the gene by removing specific sequences, and enable cells to produce functional proteins according to the corrected "blueprint," helping muscles regain vitality.
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, east China's Shanghai Jiao Tong University 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.
Xie Xiaoliang, director of Changping Laboratory and an academician of the Chinese Academy of Sciences, said that the development and application of the LEAPER platform reflects the laboratory's commitment to developing new drugs through original basic research and cutting-edge technology. He hoped that this new technology would benefit more patients suffering from major diseases. ■
