Genetic modification of the mitochondrial genome

摘要

This disclosure is in the field of genomic engineering, particularly targeted gene modification of mitochondrial DNA (mtDNA).Specifically, the methods and compositions described relate to genomic alterations (e.g., more than one or more insertions and / or deletions) through the nuclease of an endogenous mitochondrial genome (variant or wild type).A wild type of targeted tissue (e.g.Bringing phenotypic return to molecular and biochemical phenotypesWithin one or more specific tissues and / or organs (e.g.Containing the heart tissueBy nuclease mediated shift of the ratio of mutant and wild type mtDNA in the subject with mitochondrial disease.Mitochondrial genomeIt may be modified for orthodontics targeted for disease causing mutations.