Mitochondrial Genome Editing

Genome Engineering Laboratory

Mitochondrial Genome Editing

We are committed to advancing and expanding gene editing technologies for organelles, particularly to open new avenues for research and therapeutic intervention in mitochondrial genetic diseases caused by mutations in the mitochondrial genome. Over 100 pathogenic mutations in mitochondrial DNA have been associated with genetic disorders such as Leber Hereditary Optic Neuropathy (LHON) and Leigh syndrome, which currently lack effective treatments. Organellar DNA editing tools offer the potential to model these diseases in cell lines and animals and, ultimately, to correct the underlying mutations in vivo, paving the way for therapeutic applications.

Mitochondrial Genome Editing