Open access
Sep 2026
Allotopic Expression of ND6 Restores Vision in a Mitochondrial Disease Model.
This study validates the safety and therapeutic potential of allotopic expression in vivo and provides critical mechanistic insights into its role in treating LHON and other mitochondrial diseases.
Cheng Ai, Hui-Ying Li, Jing Wu et al.
· JCI Insight · 0 citations