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LHON viral vector treatment trials
IFOND scientific members, coordinating with colleagues in North America and Europe, are involved in human LHON 11778 viral vector treatment trials using technology pioneered by Dr. John Guy's team at Bascom Palmer Eye Institute at the University of Miami Miller School of Medicine in Miami Florida. Preliminary human clinical safety and efficacy trial reports have so far shown only modest benefits in restoring central vision in LHON. The technique used in the studies is to inject only one eye with active agent in a double blinded fashion per individual trial subject. Interestingly both treated and non treated eyes have similar outcomes so far. Best results were obtained in patients with early disease. See:
Zhang Y, Tian Z, Yuan J, Liu C, Liu HL, Ma SQ, Li B. The Progress of Gene Therapy for Leber's Optic Hereditary Neuropathy. Curr Gene Ther. 2017;17(4):320-326. doi: 10.2174/1566523218666171129204926. PMC5902861
Efficacy Study of GS010 for Treatment of Vision Loss From 7 Months to 1 Year From Onset in LHON Due to the ND4 Mutation (REVERSE)
Wan et al. Efficacy and Safety of rAAV2-ND4 Treatment for Leber’s Hereditary Optic Neuropathy. Sci Rep. 2016; 6: 21587. Also at http://www.nature.com/articles/srep21587
Newman NJ, Yu-Wai-Man P, Carelli V, et al. Efficacy and Safety of Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy Treated within 6 Months of Disease Onset. Ophthalmology. 2021 Jan. DOI: 10.1016/j.ophtha.2020.12.012.
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