Mitochondrial adenine base editing of mouse somatic tissues via adeno-associated viral delivery

AAV9 delivery enables mitochondrial adenine base editing in mouse tissues, achieving modest A-to-G conversions over six months.

• Why it matters: Mitochondrial genome editing has expanded potential for treating mitochondrial diseases, but in vivo editing efficiency remains limited, hindering clinical applications.
• What they did: Researchers used optimized TALEs to target mitochondrial genes in mouse cells and delivered the best pairs via systemic AAV9 to neonatal mice, measuring editing over time.
• The result: Adenine editing increased slightly from 4 weeks to 6 months, demonstrating feasibility but highlighting the need for further optimization to improve in vivo editing activity.