Researchers from Korea University (KU) led by Professor Hyunji Lee (College of Medicine) and his collaborators have engineered a precise DNA-binding protein called Transcription Activator-Like Effector (TALE)-linked deaminases – shortened to TALEDs – that will improve adenine base editing in mitochondrial DNA (mtDNA). This study could revolutionize the gene editing field, transforming the treatment of genetic diseases.

The research method explores programmable deaminases (TALEDs), proteins often used in genome editing, but combined with deaminase enzymes. To understand the magnitude of this achievement, the research paper points out how base editors – usually adenine and cytosine – are useful in research and medicine but are often limited by off-target editing in the mitochondrial genome. Nonetheless, with the new technique involving adenine-to-guanine (A-to-G) precision editing using TALEDs, KU’s research group has successfully developed the world’s first animal model with A-to-G mtDNA edits with minimal off-target mutations.

The breakthrough could bring transformative approaches for mitochondrial disorders, improving the lives of countless people affected by these diseases while maintaining the effectiveness of the editing process. Thus, making possible the development of treatments that can correct pathogenic mutations in the near future.