Mitochondrial DNA editing: Key to the treatment of neurodegenerative diseases
Neuronal death is associated with mitochondrial dysfunction caused by mutations in mitochondrial DNA. Mitochondrial DNA becomes damaged when processes such as replication, repair, and nucleotide synthesis are compromised. This extensive accumulation of damaged mitochondrial DNA subsequently disrupts...
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| Format: | Article |
| Language: | English |
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KeAi Communications Co., Ltd.
2025-07-01
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| Series: | Genes and Diseases |
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| Online Access: | http://www.sciencedirect.com/science/article/pii/S2352304224002344 |
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| author | Ye Hong Ying Song Wenjun Wang Jinghui Shi Xi Chen |
| author_facet | Ye Hong Ying Song Wenjun Wang Jinghui Shi Xi Chen |
| author_sort | Ye Hong |
| collection | DOAJ |
| description | Neuronal death is associated with mitochondrial dysfunction caused by mutations in mitochondrial DNA. Mitochondrial DNA becomes damaged when processes such as replication, repair, and nucleotide synthesis are compromised. This extensive accumulation of damaged mitochondrial DNA subsequently disrupts the normal function of mitochondria, leading to aging, degeneration, or even death of neurons. Mitochondrial dysfunction stands as a pivotal factor in the development of neurodegenerative diseases, including Parkinson's disease, Alzheimer's disease, Huntington's disease, and amyotrophic lateral sclerosis. Recognizing the intricate nature of their pathogenesis, there is an urgent need for more effective therapeutic interventions. In recent years, mitochondrial DNA editing tools such as zinc finger nucleases, double-stranded DNA deaminase toxin A-derived cytosine base editors, and transcription activator-like effector ligand deaminases have emerged. Their emergence will revolutionize the research and treatment of mitochondrial diseases. In this review, we summarize the advancements in mitochondrial base editing technology and anticipate its utilization in neurodegenerative diseases, offering insights that may inform preventive strategies and therapeutic interventions for disease phenotypes. |
| format | Article |
| id | doaj-art-35e3fa9d21cf4d26b8fb34e874fb49d4 |
| institution | OA Journals |
| issn | 2352-3042 |
| language | English |
| publishDate | 2025-07-01 |
| publisher | KeAi Communications Co., Ltd. |
| record_format | Article |
| series | Genes and Diseases |
| spelling | doaj-art-35e3fa9d21cf4d26b8fb34e874fb49d42025-08-20T02:14:24ZengKeAi Communications Co., Ltd.Genes and Diseases2352-30422025-07-0112410143710.1016/j.gendis.2024.101437Mitochondrial DNA editing: Key to the treatment of neurodegenerative diseasesYe Hong0Ying Song1Wenjun Wang2Jinghui Shi3Xi Chen4Department of Pharmacology, Zhejiang University of Technology, Hangzhou, Zhejiang 310014, ChinaDepartment of Pharmacology, Zhejiang University of Technology, Hangzhou, Zhejiang 310014, China; Hangzhou King's Bio-pharmaceutical Technology Co., Ltd., Hangzhou, Zhejiang 310007, China; Corresponding author. Department of Pharmacology, Zhejiang University of Technology, Hangzhou, Zhejiang 310014, China.Department of Pharmacology, Zhejiang University of Technology, Hangzhou, Zhejiang 310014, ChinaDepartment of Pharmacology, Zhejiang University of Technology, Hangzhou, Zhejiang 310014, ChinaDepartment of Pharmacology, Zhejiang University of Technology, Hangzhou, Zhejiang 310014, ChinaNeuronal death is associated with mitochondrial dysfunction caused by mutations in mitochondrial DNA. Mitochondrial DNA becomes damaged when processes such as replication, repair, and nucleotide synthesis are compromised. This extensive accumulation of damaged mitochondrial DNA subsequently disrupts the normal function of mitochondria, leading to aging, degeneration, or even death of neurons. Mitochondrial dysfunction stands as a pivotal factor in the development of neurodegenerative diseases, including Parkinson's disease, Alzheimer's disease, Huntington's disease, and amyotrophic lateral sclerosis. Recognizing the intricate nature of their pathogenesis, there is an urgent need for more effective therapeutic interventions. In recent years, mitochondrial DNA editing tools such as zinc finger nucleases, double-stranded DNA deaminase toxin A-derived cytosine base editors, and transcription activator-like effector ligand deaminases have emerged. Their emergence will revolutionize the research and treatment of mitochondrial diseases. In this review, we summarize the advancements in mitochondrial base editing technology and anticipate its utilization in neurodegenerative diseases, offering insights that may inform preventive strategies and therapeutic interventions for disease phenotypes.http://www.sciencedirect.com/science/article/pii/S2352304224002344Base editorCRISPR-Cas9Mitochondrial DNAmitoTALENsmitoZFNsNeurodegenerative diseases |
| spellingShingle | Ye Hong Ying Song Wenjun Wang Jinghui Shi Xi Chen Mitochondrial DNA editing: Key to the treatment of neurodegenerative diseases Genes and Diseases Base editor CRISPR-Cas9 Mitochondrial DNA mitoTALENs mitoZFNs Neurodegenerative diseases |
| title | Mitochondrial DNA editing: Key to the treatment of neurodegenerative diseases |
| title_full | Mitochondrial DNA editing: Key to the treatment of neurodegenerative diseases |
| title_fullStr | Mitochondrial DNA editing: Key to the treatment of neurodegenerative diseases |
| title_full_unstemmed | Mitochondrial DNA editing: Key to the treatment of neurodegenerative diseases |
| title_short | Mitochondrial DNA editing: Key to the treatment of neurodegenerative diseases |
| title_sort | mitochondrial dna editing key to the treatment of neurodegenerative diseases |
| topic | Base editor CRISPR-Cas9 Mitochondrial DNA mitoTALENs mitoZFNs Neurodegenerative diseases |
| url | http://www.sciencedirect.com/science/article/pii/S2352304224002344 |
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