Gene editing technology completes the last piece of the puzzle

Researchers at the Center for Genome Engineering at the Korea Institute for Basic Science (IBS) have developed a new gene editing platform called Transcription Activator-Like Effect-Associated Deaminase (TALED)

The magic and pitfalls of "gene scissors"

From the discovery of the first restriction enzyme in 1968, the invention of the polymerase chain reaction in 1985, to the demonstration of CRISPR-mediated genome editing in 2013, each new breakthrough discovery in biotechnology has further improved the ability to manipulate DNA.

While gene editing has been very successful in the nuclear genome of cells, scientists have, however, been unsuccessful in editing mitochondria, which have their own genomes

Kim Jin-soo, director of the IBS Genome Engineering Center in Korea, explained: "Due to mitochondrial DNA defects, some very serious genetic diseases have arisen

Mitochondrial DNA can be edited

The mitochondrial genome is inherited from the maternal line

"Another problem is the lack of animal models for these mitochondrial diseases

Therefore, reliable techniques for editing mitochondrial DNA are one of the frontiers of genome engineering that must be explored in order to conquer all known genetic diseases, and the world's best scientists have been working to make it a reality for years

In 2020, a research team led by the Broad Institute of Harvard University and Ruqian Liu of the Massachusetts Institute of Technology created a new base editor called DddA-derived cytosine base editor that can be extracted from DNA in mitochondria.

"We started thinking about ways to overcome these limitations," said study first author Xingyi Zhao.

The researchers created TALED by fusing three different ingredients

An interesting aspect of TALED is the ability of TadA8e to perform A to G editing in mitochondria with double-stranded DNA

Nobel Prize-level achievements

The researchers speculate that DddA tox allows access to double-stranded DNA by transiently unwinding the double-strand

The research team demonstrated the new technique by creating single-cell-derived clones containing the desired mtDNA edits

William Su, a science communicator at the Institute of Basic Sciences, praised: "I believe the significance of this discovery is comparable to the invention of the blue LED, which won the Nobel Prize in 2014.