China develops RNA editing technology for rare disease treatment
A Chinese research team has achieved a breakthrough with its newly developed RNA-editing technology, LEAPER, for treating Duchenne muscular dystrophy (DMD), a severe and rare hereditary disease. Both the details of this technology and its clinical application were published on Wednesday in the journal Cell, Qazinform News Agency cites Xinhua.
This is the first time China-developed RNA editing technology has entered clinical trials, and it is also the first global application of RNA editing for DMD treatment.
DMD is caused by gene mutations and is characterized by 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 large population. 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 for gene therapy. Its causative gene is too large for conventional gene therapy methods to deliver the full gene to patients.
In addition, different patients carry more than 7,000 types of pathogenic mutations, so a single treatment regimen can often cover only a small fraction of patients. Consequently, developing treatment strategies that are both widely applicable and long-term effective remains a significant challenge for the global medical field.
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, the LEAPER system can precisely locate the erroneous genetic "blueprint" in muscle cells, restore the gene by removing specific sequences, and enable cells to produce functional proteins based on 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 and highly safe, imposing minimal delivery burden.
The team has collaborated with Kunming University of Science and Technology in southwest China, Shanghai Jiao Tong University in eastern China, and other institutions to develop candidate drugs. All three pediatric patients who received treatment showed 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 reflect the laboratory's commitment to advancing new drug development through original basic research and cutting-edge technology. He hoped that this new technology would benefit more patients with major diseases.
Previously, Qazinform News Agency reported that as China’s population ages, Parkinson’s disease is becoming increasingly prevalent among older adults, prompting Xuanwu Hospital of Capital Medical University in Beijing to launch the country's first AI-powered digital doctor platform for the disease.
