Title of talk: Editing the mitochondrial genome: from animal models to therapy.

Talk abstract: Mutations in mitochondrial DNA (mtDNA) are a major cause of inherited retinal diseases and optic neuropathies, leading to progressive and currently untreatable visual loss. While nuclear gene editing has advanced rapidly, the ability to model and correct mtDNA mutations in the retina has remained limited. In this lecture, I will present our work establishing and validating mouse models of retinal mtDNA disease that faithfully recapitulate patient pathology, and using these models to evaluate emerging mitochondrial genome-editing technologies. We provide proof-of-concept for targeted correction of mutant mtDNA in the retina using mitochondrial base editors, and introduce the first mouse model carrying the most common Leber hereditary optic neuropathy (LHON)-causing mtDNA mutation. Together, these studies highlight the therapeutic potential of mitochondrial genome engineering and establish a translational platform for developing treatments for mtDNA-driven retinal disease.