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Mitochondrial diseases and mtDNA editing

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Mitochondrial diseases are a heterogeneous group of inherited disorders character-ized by mitochondrial dysfunction,and these diseases are often severe or even fatal.Mito-chondrial diseases are often caused by mitochondrial DNA mutations.Currently,there is no curative treatment for patients with pathogenic mitochondrial DNA mutations.With the rapid development of traditional gene editing technologies,such as zinc finger nucleases and tran-scription activator-like effector nucleases methods,there has been a search for a mitochon-drial gene editing technology that can edit mutated mitochondrial DNA;however,there are still some problems hindering the application of these methods.The discovery of the DddA-derived cytosine base editor has provided hope for mitochondrial gene editing.In this paper,we will review the progress in the research on several mitochondrial gene editing technologies with the hope that this review will be useful for further research on mitochondrial gene editing technologies to optimize the treatment of mitochondrial diseases in the future.

Gene editingMitochondrialdiseaseMitochondrial DNA mutationTranscription activator-like effector nucleasesZinc finger nucleases

Min Song、Lingqun Ye、Yongjin Yan、Xuechun Li、Xinglong Han、Shijun Hu、Miao Yu

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Department of Cardiovascular Surgery of the First Affiliated Hospital & Institute for Cardiovascular Science,Collaborative Innovation Center of Hematology,State Key Laboratory of Radiation Medicine and Protection,Suzhou Medical College,Soochow University,Suzhou,Jiangsu 215000,China

Hai'an People's Hospital,Nantong,Jiangsu 226600,China

国家重点研发计划国家重点研发计划国家自然科学基金国家自然科学基金江苏省自然科学基金中国博士后科学基金Natural Science Foundation of the Jiangsu Higher Education Institutions of ChinaJiangsu Funding Program for Excellent Postdoctoral Talent(China)Jiangsu Cardiovascular Medicine Innovation Center(China)

2022YFA11043002021YFA11019028217036482003756BK202008002022M71231221KJB3100032022ZB577CXZX202210

2024

10.1016/j.gendis.2023.06.026

基因与疾病(英文)

基因与疾病(英文)

ISSN：
年,卷(期)：2024.11(3)
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