TALEDs Gene Editing Tool Alters Mitochondrial DNA for the First Time

A research team from the College of Medicine at Korea University successfully altered the mitochondrial DNA of mice, which converts adenine (A) to guanine (G). This breakthrough is the first in history, and the paper was published in Cell.

CRISPR-Cas9 gene editing tool is commonly used for repairing DNA. However, its limitations stem from the inability of the guide RNA (gRNA) to be transported into the mitochondria. In addition, among the targeted base editing of mitochondrial DNA types, the use of transcription activator-like effector-linked deaminase (TALEDs) for A-to-G gene editing in animal studies has never been reported.

With this, the researchers engineered conventionally used TALEDs into a new variant, V28R-TALED, which could improve the precision of the DNA-modifying protein and significantly reduce unwanted DNA and RNA mutations.

Prof. Hyunji Lee, the principal investigator of the study, said, “This research marks a critical step in the journey towards therapeutic applications. We've successfully implemented an enhanced mitochondrial gene editing technique in animals, which overcomes the random DNA and RNA alterations caused by previous methods. This progress brings us closer to developing effective treatments for mitochondrial diseases, which have long lacked sufficient treatment options.”

For more information, read the article from Korea University College of Medicine.