한빛사논문
Hong Thi Lam Phan1, Hyunji Lee2,3 and Kyoungmi Kim1,4
1Department of Physiology, Korea University College of Medicine, Seoul 02841, Republic of Korea.
2Laboratory Animal Resource and Research Center, Korea Research Institute of Bioscience and Biotechnology, 28116 Cheongju, Republic of Korea.
3School of Medicine, Sungkyunkwan University, Suwon 16419, Republic of Korea.
4Department of Biomedical Sciences, Korea University College of Medicine, Seoul 02841, Republic of Korea.
Corresponding authors : Correspondence to Hyunji Lee or Kyoungmi Kim.
Abstract
Mitochondria are of fundamental importance in programmed cell death, cellular metabolism, and intracellular calcium concentration modulation, and inheritable mitochondrial disorders via mitochondrial DNA (mtDNA) mutation cause several diseases in various organs and systems. Nevertheless, mtDNA editing, which plays an essential role in the treatment of mitochondrial disorders, still faces several challenges. Recently, programmable editing tools for mtDNA base editing, such as cytosine base editors derived from DddA (DdCBEs), transcription activator-like effector (TALE)-linked deaminase (TALED), and zinc finger deaminase (ZFD), have emerged with considerable potential for correcting pathogenic mtDNA variants. In this review, we depict recent advances in the field, including structural biology and repair mechanisms, and discuss the prospects of using base editing tools on mtDNA to broaden insight into their medical applicability for treating mitochondrial diseases.
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