Fig. 3

Therapeutic approaches combining iPSCs with mtDNA gene editing technology. For iPSCs derived from patients carrying heteroplasmic mtDNA mutation, mitochondrial restriction endonucleases (mtREs), mitochondrial-targeted transcription activator-like effector nucleases (mtTALENs), and zinc-finger nucleases (mtZFN) are utilized to eliminate the mutant mtDNA molecules. For iPSCs derived from patients bearing homoplasmic mtDNA mutation, mtDNA base editing technology is preferred to correct the mutations. After gene editing, the corrected iPSCs differentiate to target cells with remodeling cellular functions. The black circles indicate mtDNA. The red dots on black circles denote mtDNA mutations. The green color of mitochondria indicates normal condition, and the pink color of mitochondria show damaged function. Other colors of cells do not indicate any information