Recent Developments in Gene Therapy for Neovascular Age-Related Macular Degeneration: A Review

Author:

Finocchio Lucia1ORCID,Zeppieri Marco1ORCID,Gabai Andrea1ORCID,Toneatto Giacomo1ORCID,Spadea Leopoldo2ORCID,Salati Carlo1

Affiliation:

1. Department of Ophthalmology, University Hospital of Udine, 33100 Udine, Italy

2. Eye Clinic, Policlinico Umberto I, “Sapienza” University of Rome, 00142 Rome, Italy

Abstract

Age-related macular degeneration (AMD) is a complex and multifactorial disease and a leading cause of irreversible blindness in the elderly population. The anti-vascular endothelial growth factor (anti-VEGF) therapy has revolutionized the management and prognosis of neovascular AMD (nAMD) and is currently the standard of care for this disease. However, patients are required to receive repeated injections, imposing substantial social and economic burdens. The implementation of gene therapy methods to achieve sustained delivery of various therapeutic proteins holds the promise of a single treatment that could ameliorate the treatment challenges associated with chronic intravitreal therapy, and potentially improve visual outcomes. Several early-phase trials are currently underway, evaluating the safety and efficacy of gene therapy for nAMD; however, areas of controversy persist, including the therapeutic target, route of administration, and potential safety issues. In this review, we assess the evolution of gene therapy for nAMD and summarize several preclinical and early-stage clinical trials, exploring challenges and future directions.

Publisher

MDPI AG

Subject

General Biochemistry, Genetics and Molecular Biology,Medicine (miscellaneous)

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