Three-year follow-up of Phase 1 and 2a rAAV.sFLT-1 subretinal gene therapy trials for exudative age related macular degeneration.

Elizabeth P Rakoczy,Aaron L Magno,Chooi-May Lai,Cora M Pierce,Mariapia A Degli-Esposti,Mark S Blumenkranz,Ian J Constable

American Journal of Ophthalmology（2019）

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摘要

•Gene therapy appeared to be well tolerated among the elderly at 36 months.•The presence of anti-AAV2 neutralizing antibodies at baseline did not seem to interfere with the outcome.•Patients (HD-1) who responded at 12 months to gene therapy did well over the next 24 months.

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关键词

AAV,AMD,CNV,choroidal neovascularisation,long-term,recombinant adeno-associated virus,retina,sFLT-1,subretinal injection,vascular endothelial growth factor,wet age-related macular degeneration

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