A research team from the School of Biomedical Sciences, LKS Faculty of Medicine, University of Hong Kong (HKUMed) has achieved a significant biotechnological advance that could revolutionize treatment strategies for neurodegenerative diseases. The research team has developed a new tool called RNA segment editing (RSE) that works like a “cut-and-patch” tool for RNA. This innovative approach allows scientists to precisely remove or replace defective segments of genetic messages within living cells without permanently altering a person’s DNA. This discovery paves the way for a new generation of reversible targeted therapies for neurodegenerative diseases such as Huntington’s disease. This study was published in a prestigious journal Nature Communications.
Targeted RNA repair: fix errors without damaging cells
While DNA is often referred to as the “blueprint” of life, RNA is the messenger that carries instructions for building essential proteins within cells. “In many genetic diseases, such as Huntington’s disease and certain cancers, the RNA message may contain disease-causing errors or toxic segments,” said Professor Kwon Sung-cheol, assistant professor in the School of Biomedical Sciences at the University of Hong Kong. Current editing tools often destroy the entire RNA message or modify only a single letter, severely limiting therapeutic potential. Correcting these RNA errors has been a long-standing challenge.
To overcome this challenge, the research team focused on an enzyme called Cas13, which acts as a molecular scissor that targets RNA rather than DNA. By manipulating Cas13, the team achieved much greater precision, allowing them to “cut” the RNA at precise locations.
Based on this discovery, the team developed an RSE platform that acts like a “search and replace” function for long segments of RNA. It cuts out the “defective” part of the message and repairs it with a healthy “patch” of living cells, providing a targeted approach to correcting disease-causing genetic messages.
Restore function in Huntington’s disease
The potential effects of this “patch” are significant for neurodegenerative diseases such as Huntington’s disease. In this condition, toxic repetitive segments within the RNA cause brain cells to malfunction. Current experimental treatments often delete the entire RNA message, risking loss of beneficial protein function. The innovative RSE approach can selectively remove harmful segments while preserving healthy segments of RNA.
Professor Kwon Sung-chul said:Our goal is to create tools that enable programmable RNA repair without permanently altering a patient’s DNA. RSE offers a flexible and safe approach that can be tailored to treat neurodegenerative diseases. This opens up exciting new possibilities for RNA-based therapies, which can be adjusted or reversed by simply stopping treatment, similar to traditional pills. ”
sauce:
University of Hong Kong
Reference magazines:
Ram, J.K.C.et al. (2026). Molecular basis of target RNA cleavage by Cas13. nature communications. DOI: 10.1038/s41467-026-71578-7. https://www.nature.com/articles/s41467-026-71578-7
