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Gene Therapy for Leber Hereditary Optic Neuropathy: Low- and Medium-Dose Visual Results.
Guy John, Feuer William J, Davis Janet L, Porciatti Vittorio, Gonzalez Phillip J, Koilkonda Rajeshwari D et al.
OphthalmologyJun 2017168 citationsClinical Trial
This study found low/medium-dose gene therapy for LHON was safe and improved vision in treated eyes, suggesting promise for further testing of higher doses.