Gene Therapy for Choroidal Neovascularization

초록

The most common reason for untreatable serious loss of vision in patients aged 55+ in the developed world is age-related macular degeneration (AMD). A primary cause of serious vision loss related to AMD is choroidal neovascularization (CNV) related to neurovascular AMD (nAMD). nAMD typically presents with abnormalities in vessels caused by underlying choriocapillaris. It is common for newly created blood vessels to be leaky, which can cause irreversible damage to sensitive photoreceptor cells. A full understanding of the molecular causes of nAMD has yet to be attained; the cause of the disease and its progression are attributable to numerous causes, which include genetics, ethnicity, age, and diet. A number of growth factors are believed to be involved with the condition, vascular endothelial growth factor (VEGF) among them. Clinical outcomes have been considerably improved with the arrival of anti-VEGF therapy. In clinical trials, drug therapies have returned positive outcomes, but on a wider scale results have not been as impressive, even in environments where healthcare standards are high. As well as having limited therapeutic value, patients on these drug therapies must be injected every month for the rest of their lifetime for the benefits to continue, and so it is clear that improved therapies are required. With genetic retinal disorders, e.g., choroideremia, Leber’s congenital amaurosis, and retinitis pigmentosa, the potential for gene therapy has been explored; in recent times, its potential for treating AMD has also been investigated. This chapter will examine the newest advances in AMD gene therapy and offer a summary of those approved clinical trials that have tested gene therapy as an nAMD treatment. © Springer Nature Singapore Pte Ltd. 2020.

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