Table 2 Gene therapy
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–angiotensin 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) |