Repurposing small RNA from ciliates for genome editing: single-molecule study

The “MIGHTY_RNA” project addressed the critical need for more precise and efficient genome editing techniques, which are essential for advancing the understanding and treatment of genetic disorders, cancers, and other diseases. Improved genome editing methods are vital for society as they hold the potential to revolutionize healthcare, enhance food security, and foster innovations in personalized medicine, ultimately improving the quality of life and driving economic growth. The project aimed to elucidate the molecular mechanisms of DNA modification and apply these insights to genome editing. Specifically, it sought to elucidate the functional modes of Argonaute-mediated target recognition by developing a new single-molecule technique named SPARXS and to expand the study to include other genome editing systems.

From the outset, the project focused on developing innovative methodologies and exploring new genome editing systems. The high throughput single-molecule fluorescence technique, SPARXS, was successfully developed, significantly enhancing the capacity for precise molecular studies. This breakthrough was well-documented and accepted by leading scientific journals, including Science. This research expanded to include the examination of other novel proteins, using single-molecule FRET to understand their functional aspects. These studies have been published in high-impact journals like Nature Communications, providing valuable insights into genome editing. Throughout the project, the findings were disseminated through seminars, international conferences, and publications, promoting knowledge transfer.

The project has significantly advanced the state of the art in genome editing. The development of SPARXS represents a major breakthrough, enabling high-throughput and precise observations of molecular interactions, with wide-ranging applications in medical diagnostics and therapeutic development. The exploration has opened new avenues for genome editing research, providing new insights into novel proteins’ biophysics and potential applications. These advancements are expected to continue driving innovation in genome editing, positioning Europe to lead future research and foster collaborations. Ultimately, the project’s achievements contribute to a future where genetic diseases can be better understood and treated, improving health outcomes and quality of life globally.