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Journal: Optimal Inhibition of Choroidal Neovascularization by scAAV2 with VMD2 Promoter-driven Active Rap1a in the RPE. (Pubmed Central) - Nov 7, 2020
scAAV2-VMD2, but not scAAV2-RPE65, specifically and efficiently transduced the RPE to increase active Rap1a protein in the RPE...Increased active Rap1a in the RPE in vivo or in vitro inhibited inflammatory and angiogenic signaling determined by decreased activation of NF-κB and expression of VEGF without causing increased cell death or autophagy measured by increased LCA3/B. Our study provides a potential future strategy to deliver active Rap1a to the RPE in order to protect against both atrophic and neovascular AMD.
- |||||||||| AAV2-RPE65 / Medical Research Council, University College London, MeiraGTx, Luxturna (voretigene neparvovec-rzyl) / Novartis, Roche, rAAV2-CB-hRPE65 / AGTC
Journal: Results at 5 Years after Gene Therapy for RPE65-deficient Retinal Dystrophy. (Pubmed Central) - Jun 18, 2020
There were no clinically significant adverse events. Visual acuity and static perimetry testing results suggest that treating patients at a younger age is associated with better visual function outcomes during 5 years after treatment.
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Trial completion, P1/2 data, Trial initiation date: Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis (clinicaltrials.gov) - Dec 20, 2017
P1/2, N=12, Completed,
Visual acuity and static perimetry testing results suggest that treating patients at a younger age is associated with better visual function outcomes during 5 years after treatment. Active, not recruiting --> Completed | Initiation date: Dec 2008 --> Jun 2009
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