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The rAAV2-ND1 gene therapy for Leber hereditary optic neuropathy.
Li Xin, Yuan Jun, Qi Jia, Ruan Kunwei, Li Bin, Dan Yanping et al.
Graefes Arch Clin Exp OphthalmolFeb 20253 citationsClinical Trial
This study found rAAV2-ND1 gene therapy for ND1-LHON was safe and improved vision in treated eyes, with some benefit in untreated eyes, offering a promising new treatment.