In vivo mitochondrial base editing via adeno-associated viral delivery to mouse post-mitotic tissue.

ABSTRACT: Mitochondria host key metabolic processes vital for cellular energy provision and are central to cell fate decisions. They are subjected to unique genetic control by both nuclear DNA and their own multi-copy genome - mitochondrial DNA (mtDNA). Mutations in mtDNA often lead to clinically heterogeneous, maternally inherited diseases that display different organ-specific presentation at any stage of life. For a long time, genetic manipulation of mammalian mtDNA has posed a major challenge, impeding our ability to understand the basic mitochondrial biology and mechanisms underpinning mitochondrial disease. However, an important new tool for mtDNA mutagenesis has emerged recently, namely double-stranded DNA deaminase (DddA)-derived cytosine base editor (DdCBE). Here, we test this emerging tool

SUBMITTER: Silva-Pinheiro P 

PROVIDER: S-EPMC8825850 | biostudies-literature | 2022 Feb

REPOSITORIES: biostudies-literature