Precision Gene Therapy: A Transformative Approach to Treat Age-Related Macular Degeneration (ARMD)

Abstract

A new age of Precision gene therapy is the most futuristic, transformative, and alternative approach developed for treating Age-Related Macular Degeneration (ARMD). Globally, ARMD is a major contributor to severe, irreversible vision loss. It is a degenerative disease that results in central vision loss by affecting the choriocapillaris, photoreceptors, retinal pigment epithelium, and macula, thereby limiting the frequency of anti-vascular endothelial growth factor (anti-VEGF) injections as a standard treatment. Additionally, it involves creating techniques to ensure sustained delivery of a range of anti-angiogenic proteins. These are repetitive intravitreal injections, which put the patient at high risk of infection, costly drugs, low compliance, disparities in access, healthcare burden, increase the doctor-patient conflict, and are hectic for the elderly population. Gene therapy has emerged as a promising substitute, an innovative approach to treating ARMD by replacing faulty genes with healthy ones. Viral non-integrating vectors, such as Adeno-Associated Viral (AAV), act as envelopes that carry encoded genetic messages without impacting native cellular DNA. This Review aims to provide a comprehensive overview of gene therapy for patients with ARMD, providing the current status of ongoing research, awareness, and future innovative progression. The high potential and efficiency of this therapy could make it an effective method, providing hope for patients and potentially revolutionizing the future of healthcare.