线粒体基因组编辑：策略、挑战和应用。

IF 2.9 3区生物学 Q3 BIOCHEMISTRY & MOLECULAR BIOLOGY

BMB Reports Pub Date : 2024-01-04 DOI:10.5483/bmbrep.2023-0224

Kayeong Lim

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引用次数: 0

摘要

线粒体 DNA（mtDNA）是线粒体中的多拷贝基因组，对氧化磷酸化至关重要。mtDNA 突变可导致能量需求高的组织和器官出现严重的线粒体功能障碍。mtDNA突变与线粒体疾病和年龄相关疾病密切相关。为了更好地了解 mtDNA 的功能作用并开发治疗方法，必须推进能够操纵线粒体基因组的技术。本综述将讨论目前使用 mtDNA 核酸酶和碱基编辑器进行线粒体基因组编辑的工作，包括工具、传输策略和应用。线粒体基因组编辑的未来进展将解决其效率和特异性方面的挑战，从而实现治疗性基因组编辑的承诺。

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Mitochondrial genome editing: strategies, challenges, and applications.

Mitochondrial DNA (mtDNA), a multicopy genome found in mitochondria, is crucial for oxidative phosphorylation. Mutations in mtDNA can lead to severe mitochondrial dysfunction in tissues and organs with high energy demand. MtDNA mutations are closely associated with mitochondrial and age-related disease. To better understand the functional role of mtDNA and work toward developing therapeutics, it is essential to advance technology that is capable of manipulating the mitochondrial genome. This review discusses ongoing efforts in mitochondrial genome editing with mtDNA nucleases and base editors, including the tools, delivery strategies, and applications. Future advances in mitochondrial genome editing to address challenges regarding their efficiency and specificity can achieve the promise of therapeutic genome editing.

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来源期刊

BMB Reports 生物-生化与分子生物学

CiteScore

5.10

自引率

7.90%

发文量

141

审稿时长

1 months

期刊介绍： The BMB Reports (BMB Rep, established in 1968) is published at the end of every month by Korean Society for Biochemistry and Molecular Biology. Copyright is reserved by the Society. The journal publishes short articles and mini reviews. We expect that the BMB Reports will deliver the new scientific findings and knowledge to our readers in fast and timely manner.