Research

1.     Addressing Unresolved Challenges in Gene Editing

• We are committed to tackling the challenges that have hindered the full potential of gene editing. This includes exploring novel approaches and strategies to overcome existing limitations, aiming for solutions that have yet to be achieved in the field of gene correction. Through these efforts, we strive to develop more reliable and accurate tools for gene editing that can address previously unsolved issues. 

ex) In 2022, we developed the world’s first mitochondrial adenine base editor, TALED. This was a significant achievement because half of mitochondrial diseases can be corrected using adenine base editor. However, until then, no mitochondrial adenine base editor existed, and we recognized the need for such technology. In 2024, we developed an enhanced TALED that addresses off-target effects, further advancing the technology.

2.     Advancing and Refining genome editing technologies, both for nuclear and mitochondrial genomes. 

• Nuclear Genome Editing: Nucleases (ZFN, TALEN, CRISPR-Cas9), base editors (ABE, CBE), prime editors, Large gene insertion, etc.

• Mitochondrial Genome Editing: Mitochondria-specific nucleases (mitoZFN, mitoTALEN), base editors (mitoABE-TALED, mitoCBE-DdCBE, ZFD).

Our goal is to enhance the effectiveness of these already developed technologies, making them more precise and efficient for use in various applications.

3.     Applications (Therapeutics)

• Genome Editing for Major Diseases: We apply genome editing technologies to address significant human diseases, including cancer, neurodegenerative disorders, etc.

• Aging and Biological Processes: Our research investigates the molecular mechanisms underlying aging and other biological processes, exploring how gene editing can improve our understanding and contribute to advancements in these areas.

• Therapeutics for Rare Diseases: We focus on developing gene therapies for mitochondrial diseases (e.g., LHON, Leigh Syndrome, MELAS) and amyotrophic lateral sclerosis (ALS), offering new therapeutic options for rare and challenging conditions.