- Protein Engineering for Better Performance in Genome Engineering
- Mouse Model for Genetic Disease
- Therapeutic Mitochondrial Genome Editing
Our group is researching cutting-edge biotechnology related to mitochondria and aiming to develop novel genome editing proteins for mitochondrial science. Mitochondrial DNA genome editing offers a remarkable opportunity to create animal models for human genetic disorders resulting from problematic mutations within the mitochondrial genome. Moreover, it holds promise for potential therapies using tools like DdCBEs, ZFD, TALED, and mitoBE, which involve DNA-binding proteins and DNA-modifying components like deaminase.
However, the practical use of mitochondrial base editors faces challenges like selectivity in sequences, unintended editing effects, and off-target impacts on both mitochondrial and nuclear DNA. Our team is dedicated to refining genome editing techniques through advanced protein engineering. This improvement in DNA editing tools is crucial for achieving effective genome modifications. Additionally, our goal involves generating mice with edited mitochondrial DNA carrying specific mitochondrial diseases, and exploring potential therapeutic mitochondrial DNA editing. By utilizing reliable tools to introduce point mutations in mouse embryos, we can explore the consequences of various mitochondrial DNA mutations.
Through our research, we aim to surpass these challenges and expand our insights into mitochondrial genetics and associated disorders. Our commitment is to pioneer innovative genome editing techniques that have the potential to transform mitochondrial medicine and disease research.
1. "Comprehensive phenotypic assessment of mitochondrial ND5 nonsense mutation in mice", Experimental and Molecular Medicine, 2024, in press, *Co-correspondence
2. "Engineering TALE-linked deaminases to facilitate precision adenine base editing in mitochondrial DNA", Cell, 2024, 187(1), 95-106, e26, 1/4, https://doi.org/10.1016/j.cell.2023.11.035, *Co-correspondence
3. “Precision mitochondrial DNA editing with high-fidelity DddA-derived base editors”, Nature Biotechnology, 2022, 41, 378-386, 10.1038/ s41587-022-01486-w, IF = 46.9
4. “Enhanced mitochondrial DNA editing in mice using nuclear-exported TALE-linked deaminases and nucleases”, Genome Biology, 2022, 23, 211, 10.1186/ s13059-022-02782-z, IF = 12.3
5. “Mitochondrial DNA editing in mice with DddA-TALE fusion deaminases”, Nature Communications, 2021, 12, 1190, IF = 17.7
(Tel) +82-31-299-6185,
(Email) shlee9@skku.edu