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线粒体碱基编辑:治疗机会的最新进展。

Mitochondrial Base Editing: Recent Advances towards Therapeutic Opportunities.

机构信息

Department of Biochemistry and Molecular Biology, Mayo Clinic, Rochester, MN 55905, USA.

Mayo Clinic Graduate School of Biomedical Sciences, Virology and Gene Therapy Track, Mayo Clinic, Rochester, MN 55905, USA.

出版信息

Int J Mol Sci. 2023 Mar 18;24(6):5798. doi: 10.3390/ijms24065798.

Abstract

Mitochondria are critical organelles that form networks within our cells, generate energy dynamically, contribute to diverse cell and organ function, and produce a variety of critical signaling molecules, such as cortisol. This intracellular microbiome can differ between cells, tissues, and organs. Mitochondria can change with disease, age, and in response to the environment. Single nucleotide variants in the circular genomes of human mitochondrial DNA are associated with many different life-threatening diseases. Mitochondrial DNA base editing tools have established novel disease models and represent a new possibility toward personalized gene therapies for the treatment of mtDNA-based disorders.

摘要

线粒体是细胞内的重要细胞器,它们形成网络,动态产生能量,有助于细胞和器官的多种功能,并产生各种关键的信号分子,如皮质醇。这种细胞内微生物组在不同的细胞、组织和器官之间存在差异。线粒体可以随着疾病、年龄和环境的变化而变化。人类线粒体 DNA 环状基因组中的单核苷酸变异与许多不同的危及生命的疾病有关。线粒体 DNA 碱基编辑工具已经建立了新的疾病模型,为基于 mtDNA 疾病的个性化基因治疗提供了新的可能性。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/05e5/10056815/e898ec500b59/ijms-24-05798-g001.jpg

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