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- W2983538863 abstract "Abstract Mitochondrial DNA (mtDNA) disorders include a wide range of pathological conditions, sharing the inability to produce adenosine triphosphate efficiently. These disorders are among the most prevalent of all genetic disorders. The development of gene therapy to treat mtDNA disorders offers a promising approach, as it acts in the actual cause of the disorder. Some researchers have devoted their attention to the design of suitable mtDNA-based delivery systems, and significant progress has been accomplished in the mitochondrial gene therapy field. As a result, and by different approaches, today several mtDNA carriers can target and deliver genetic material into mitochondria, highlighting the feasibility of this therapy for future clinical translation. In line with this, mitochondrial-targeted plasmid DNA nanoparticles have been developed by incorporation of rhodamine 123, a fluorescent amphiphile with mitochondria affinity. The pDNA vectors were easily internalized into different cell lines and can target delivery to mitochondria. The acquired knowledge instigates further research and clinical translation." @default.
- W2983538863 created "2019-11-22" @default.
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- W2983538863 date "2019-01-01" @default.
- W2983538863 modified "2023-10-16" @default.
- W2983538863 title "Development of plasmid DNA nanoparticles for mitochondrial gene therapy" @default.
- W2983538863 doi "https://doi.org/10.1016/b978-0-12-818435-6.00011-6" @default.
- W2983538863 hasPublicationYear "2019" @default.
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