Mitochondrial genome editing

Prof. Wensheng Wei published a review in Nature Reviews Bioengineering.

Mitochondria possess small, maternally inherited genomes that encode indispensable components of the oxidative phosphorylation machinery. Mitochondrial DNA (mtDNA) mutations underlie a broad spectrum of metabolic and degenerative disorders, yet therapeutic options remain limited. Advances in mitochondrial genome engineering, including programmable nucleases and mitochondria-compatible base editors, enable the selective depletion of mutant genomes and the installation of defined C-to-T and A-to-G substitutions. These technologies have enabled the development of cellular and animal models for mechanistic studies of mitochondrial biology and opened up avenues for potential therapeutic intervention. In this Review, we discuss the development and application of current mtDNA-editing platforms and examine the key technical barriers and opportunities for their clinical translation.