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Table 2 Gene therapy

From: Novel stem cell and gene therapy in diabetic retinopathy, age related macular degeneration, and retinitis pigmentosa

Condition Goal Method
Diabetic retinopathy Reduction of angiogenesis Downregulation of VEGF via gene targeting of sFlt-1, Flt23k, and PEDF
Reduction of oxidative stress Vector-mediated delivery of superoxide dismutase
Regulation of renin‚Äďangiotensin system Targeting genes of ACE 2, Ang, or Mas receptor
Macular degeneration Inhibition of angiogenesis and neovascularization Binding of VEGF by proteins delivered by AAV2 vectors
AAV2 vectors carrying sFLT-1
Viral vectors carrying endostatin and angiostatin
Retinitis pigmentosa Decreased loss of photoreceptors and preserved retinal function due to improved phagocytic function Vector delivery of MERTK gene
Increased cell survival Increased expression of GDNF
Restore proper expression of the RPGR gene Production of the retinitis pigmentosa GTPase regulator gene (commonly mutated in X-linked retinitis pigmentosa)