Gene Therapy Research Center, Digestive Diseases Research Institute, Shariati Hospital, Tehran University of Medical Sciences, Tehran, Iran.
J Ocul Pharmacol Ther. 2022 Oct;38(8):529-548. doi: 10.1089/jop.2022.0022. Epub 2022 Sep 19.
Neovascularization in ocular vessels causes a major disease burden. The most common causes of choroidal neovascularization (CNV) are age-related macular degeneration and diabetic retinopathy, which are the leading causes of irreversible vision loss in the adult population. Vascular endothelial growth factor (VEGF) is critical for the formation of new vessels and is the main regulator in ocular angiogenesis and vascular permeability through its receptors. Laser therapy and antiangiogenic factors have been used for CNV treatment. Bevacizumab, ranibizumab, and aflibercept are commonly used anti-VEGF agents; however, high costs and the need for frequent intraocular injections are major drawbacks of anti-VEGF drugs. Gene therapy, given the potency of one-time treatment and no need for frequent injections offers the real possibility of such a lasting treatment, with fewer adverse effects and higher patient quality of life. Herein, we reviewed the role of gene therapy in the CNV treatment. In addition, we discuss the advantages and challenges of current treatments compared with gene therapy.
眼血管新生导致了重大的疾病负担。脉络膜新生血管(CNV)最常见的病因是年龄相关性黄斑变性和糖尿病性视网膜病变,这两种疾病是成年人不可逆视力丧失的主要原因。血管内皮生长因子(VEGF)对于新血管的形成至关重要,它通过其受体成为眼血管生成和血管通透性的主要调节剂。激光治疗和抗血管生成因子已被用于 CNV 的治疗。贝伐单抗、雷珠单抗和阿柏西普是常用的抗 VEGF 药物;然而,高成本和频繁的眼内注射是抗 VEGF 药物的主要缺点。基因治疗由于单次治疗的效力和无需频繁注射,为这种持久治疗提供了真正的可能性,具有更少的不良反应和更高的患者生活质量。在此,我们综述了基因治疗在 CNV 治疗中的作用。此外,我们还讨论了与基因治疗相比,目前治疗方法的优势和挑战。