Revolutionary RNA Editing Tools Promise Safer Genetic Therapies, Pioneered by Yale Scientists
August 21, 2025
Researchers have developed new RNA editing tools, R-IscB and R-Cas9, which can perform versatile genetic interventions at the RNA level, including trans-splicing reactions to correct mutations.
Published in the journal Cell, the research demonstrates that these tools can target and modify RNA, offering promising avenues for treating genetic diseases and promoting wound healing.
These innovative tools leverage a previously hidden RNA-targeting activity within the CRISPR-associated enzyme IscB, uncovered through detailed molecular structural analysis, making the editing process more precise and less toxic.
The discovery of this RNA-targeting activity inside IscB enhances the specificity of the editing process, reducing potential side effects and expanding the possibilities for genetic therapies.
The research was conducted by Yale scientists in collaboration with the University of Chicago, providing new pathways for safer and more accurate genetic treatments.
Led by Yale researchers including senior author Ailong Ke and first author Chengtao Xu, the study confirms that these tools work effectively on Cas9 targets guided by CRISPR sequences.
Yale researchers have created a new family of RNA editing tools, which target messenger RNA (mRNA) instead of DNA, thereby reducing the risk of permanent heritable genetic changes.
These versatile tools can perturb, slice, destroy, or correct mRNA, functioning as 'Swiss army knives' for RNA editing, and opening new possibilities in genome medicine.
This breakthrough provides a robust, precise, and versatile RNA editing technology that could mitigate risks associated with traditional DNA editing.
The researchers emphasize the potential of trans-splicing reactions enabled by R-IscB to correct various genetic mutations at the RNA level, marking a major advance in genome medicine.
Future plans include testing these tools in the lab to treat rare genetic diseases and enable advanced genetic corrections, demonstrating their promising therapeutic potential.
Summary based on 2 sources
Get a daily email with more Science stories
Sources

Phys.org • Aug 18, 2025
RNA editing tool can take some of the risk out of gene therapy
News-Medical • Aug 21, 2025
Yale researchers uncover new RNA editing tools for safer genetic surgery