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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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The information contained on this website should not be considered medical guidance or professional advice. IFOND is not responsible for errors or omissions in information provided on this site or actions resulting from its use. IFOND does not publish all information from all available sources on optic nerve disease. IFOND is not responsible for the validity of the studies or reviews nor is it an advocate of studies or reviews mentioned on or linked from the IFOND web site. IFOND does not endorse or recommend participation in any particular clinical trial or treatment protocol which may be mentioned on this site. Direct any questions concerning your personal health to your appropriate health care professional.