en.Wedoany.com Reported - A team led by Wei Wensheng, a leading scientist at Changping Laboratory and a professor at Peking University, has developed a novel RNA exon-skipping therapy based on their independently developed RNA editing technology, LEAPER. This therapy has demonstrated positive efficacy in clinical studies for Duchenne muscular dystrophy (DMD), a rare genetic disorder. Two related research findings were simultaneously published on June 10 in the international academic journal Cell.
Unlike traditional gene editing, LEAPER technology directly utilizes naturally occurring RNA editing enzymes within human cells, eliminating the need to deliver exogenous editing proteins. The team designed a "recognize-skip-restore" therapeutic strategy that regulates RNA processing to prompt cells to regenerate functional proteins. This technological system reduces the difficulty of delivering exogenous genes or proteins into the body, better meets clinical research requirements, and creates conditions for the rapid transition of the technology from basic research to clinical application.
The pathogenic gene for DMD is exceptionally large, making it difficult for traditional gene therapy methods to deliver the complete gene into patients. Moreover, different patients carry over 7,000 types of pathogenic mutations. The exon-skipping strategy shifts gene therapy from single-point repair to group-level correction, enhancing the system's adaptability.
Based on this technology, the team, in collaboration with domestic research and medical institutions, completed a systematic translational study from disease model validation to patient treatment, developing the RNA editing drug candidate LE051. One-year follow-up results showed that all treated patients exhibited sustained improvements in motor function.
The team also utilized artificial intelligence structural prediction technology to, for the first time, systematically reveal key structural principles governing how RNA editing enzymes recognize and edit RNA. Based on these findings, they developed the next-generation RNA editing technology, LEAPER 3.0, which will drive the development of more safe, universal, and precisely controllable novel RNA therapeutic strategies in the future.
Xie Xiaoliang, director of Changping Laboratory and an academician of the Chinese Academy of Sciences, stated that the achievements of Wei Wensheng's team exemplify how the laboratory leverages original basic research and cutting-edge technology development to advance new drug discovery, expressing hope that this foundational technology can bring new treatment options for patients with more major diseases.
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