Trends and prospects in mitochondrial genome editing

Hong Thi Lam Phan, Hyunji Lee, Kyoungmi Kim

Research output: Contribution to journalReview articlepeer-review

5 Citations (Scopus)


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.

Original languageEnglish
Pages (from-to)871-878
Number of pages8
JournalExperimental and Molecular Medicine
Issue number5
Publication statusPublished - 2023 May

Bibliographical note

Funding Information:
This study was supported by the Chung Yang, Cha Young Sun, and Jang Hi Joo Memorial Fund. This work was also supported by the Bio & Medical Technology Development Program of the National Research Foundation (NRF) of Korea (NRF-2020M3A9D5A01082439 and NRF-2019R1A2C2087198) and the Korea Research Institute of Bioscience and Biotechnology (KRIBB) Research Initiative Program. The illustrations were generated using

Publisher Copyright:
© 2023, The Author(s).

ASJC Scopus subject areas

  • Biochemistry
  • Molecular Medicine
  • Molecular Biology
  • Clinical Biochemistry


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