Abstract
Age-related macular degeneration (AMD) is a degenerative eye disease that is the leading cause of irreversible vision loss in people 50 years and older. Today, the most common treatment for AMD involves repeated intravitreal injections of anti-vascular endothelial growth factor (VEGF) drugs. However, the existing expensive therapies not only cannot cure this disease, they also produce a variety of side effects. For example, the number of injections increases the cumulative risk of endophthalmitis and other complications. Today, a single intravitreal injection of gene therapy products can greatly reduce the burden of treatment and improve visual effects. In addition, the latest innovations in nanotherapy provide the best drug delivery alternative for the treatment of AMD. In this review, we discuss the development of nano-drug delivery systems and gene therapy strategies for AMD in recent years. In addition, we discuss some novel targeting strategies and the potential application of these delivery methods in the treatment of AMD. Finally, we also propose that the combination of CRISPR/Cas9 technology with a new non-viral delivery system may be promising as a therapeutic strategy for the treatment of AMD.
Highlights
Age-related macular degeneration (AMD), an eye disease with irreversible vision loss caused by retinal epithelial cell decay and retinal degeneration, is currently the third leading cause of blindness among the elderly [1]
Since anti-vascular endothelial growth factor (VEGF) therapy was used in clinics, researchers have been looking for better treatment regimens, and gene therapy is expected to be a better alternative to long-term anti-VEGF treatment
The results show that RAC-derived exosomes can target macrophages and vascular endothelial cells involved in the formation of choroidal neovascularization (CNV), and inhibit laser-induced CNV and retinal vascular leakage
Summary
Age-related macular degeneration (AMD), an eye disease with irreversible vision loss caused by retinal epithelial cell decay and retinal degeneration, is currently the third leading cause of blindness among the elderly [1]. Due to the special structure of the eye as a natural barrier to drug absorption, local non-invasive ocular drug delivery for anterior chamber diseases is not suitable for AMD. Compared with other local administration methods, the drug is injected into the vicinity of the retinal tissue to produce the highest level of bioavailability in the posterior chamber group. Vascular endothelial growth factor (VEGF) inhibitors are mostly used clinically to treat wAMD, including the intravitreal injection of ranibizumab, VEGF-Trap (Aflibercept injection) and the injection of the off-label drug bevacizumab (Table 1). These antibody drugs can significantly improve the visual prognosis of patients with wAMD, some patients will show resistance to antiVEGF inhibitors. The structure of the eye, the larger molecular weight, and anti-VEGF antibody drug efficacy being shorter, requires invasive injection through the vitreous cavity
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