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线粒体移植:一种有前途的治疗方法概述。

Mitochondrial transplantation: an overview of a promising therapeutic approach.

机构信息

Metabolic Regulation Research Center, Korea Research Institute of Bioscience and Biotechnology, Daejeon 34141; Department of Functional Genomics, University of Science and Technology (UST) of Korea, Daejeon 34113, Korea.

Department of Functional Genomics, University of Science and Technology (UST) of Korea, Daejeon 34113; Biodefense Research Center, Korea Research Institute of Bioscience and Biotechnology, Daejeon 34141, Korea.

出版信息

BMB Rep. 2023 Sep;56(9):488-495. doi: 10.5483/BMBRep.2023-0098.

Abstract

Mitochondrial transplantation is a promising therapeutic approach for the treatment of mitochondrial diseases caused by mutations in mitochondrial DNA, as well as several metabolic and neurological disorders. Animal studies have shown that mitochondrial transplantation can improve cellular energy metabolism, restore mitochondrial function, and prevent cell death. However, challenges need to be addressed, such as the delivery of functional mitochondria to the correct cells in the body, and the long-term stability and function of the transplanted mitochondria. Researchers are exploring new methods for mitochondrial transplantation, including the use of nanoparticles or CRISPR gene editing. Mechanisms underlying the integration and function of transplanted mitochondria are complex and not fully understood, but research has revealed some key factors that play a role. While the safety and efficacy of mitochondrial transplantation have been investigated in animal models and human trials, more research is needed to optimize delivery methods and evaluate long-term safety and efficacy. Clinical trials using mitochondrial transplantation have shown mixed results, highlighting the need for further research in this area. In conclusion, although mitochondrial transplantation holds significant potential for the treatment of various diseases, more work is needed to overcome challenges and evaluate its safety and efficacy in human trials. [BMB Reports 2023; 56(9): 488-495].

摘要

线粒体移植是一种有前途的治疗方法,可用于治疗由线粒体 DNA 突变引起的线粒体疾病,以及多种代谢和神经疾病。动物研究表明,线粒体移植可以改善细胞能量代谢,恢复线粒体功能,防止细胞死亡。然而,需要解决一些挑战,例如将功能性线粒体递送到体内正确的细胞,以及移植线粒体的长期稳定性和功能。研究人员正在探索新的线粒体移植方法,包括使用纳米粒子或 CRISPR 基因编辑。移植线粒体的整合和功能的机制很复杂,尚未完全理解,但研究揭示了一些起作用的关键因素。虽然线粒体移植在动物模型和人体试验中的安全性和有效性已经得到了研究,但仍需要更多的研究来优化递送方法并评估其长期安全性和疗效。使用线粒体移植的临床试验结果喜忧参半,突出表明需要在该领域进行进一步研究。总之,尽管线粒体移植在治疗各种疾病方面具有重要潜力,但仍需要克服挑战,并在人体试验中评估其安全性和疗效。[BMB 报告 2023;56(9):488-495]。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7930/10547968/0b085c65a635/bmb-56-9-488-f1.jpg

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