New potentials of mitochondrial DNA editing

Abstract

Gene modification and editing using mitochondrial DNA (mtDNA) is a promising option to conventional therapy for mitochondrial dysfunction which can result in a wide range of diseases and failure of organ and tissue functions. RNA-free DddA-derived cytosine base editors have been a promising method due to its high target specificity and high product purity in human mtDNA. However, mtDNA editing is still only a promising option and further studies and investigations are required prior to implementing it in different cell types, organelles, and various diseases as a new clinical therapy.