Review of gene therapies for age-related macular degeneration

Arshad M. Khanani, Mathew J. Thomas, Aamir A. Aziz, Christina Y. Weng, Carl J. Danzig, Glenn Yiu, Szilárd Kiss, Nadia K. Waheed, Peter K. Kaiser

Research output: Contribution to journalReview articlepeer-review

1 Scopus citations


Gene therapies aim to deliver a therapeutic payload to specified tissues with underlying protein deficiency. Since the 1990s, gene therapies have been explored as potential treatments for chronic conditions requiring lifetime care and medical management. Ocular gene therapies target a range of ocular disorders, but retinal diseases are of particular importance due to the prevalence of retinal disease and the current treatment burden of such diseases on affected patients, as well as the challenge of properly delivering these therapies to the target tissue. The purpose of this review is to provide an update on the most current data available for five different retinal gene therapies currently undergoing clinical trials for use against age-related macular degeneration (AMD) and the development of novel delivery routes for the administration of such therapies. Research has been performed and compiled from PubMed and the select authors of this manuscript on the treatment and effectiveness of five current retinal gene therapies: Luxturna, ADVM-022, RGX-314, GT-005, and HMR59. We present the available data of current clinical trials for the treatment of neovascular and dry age-related macular degeneration with different AAV-based gene therapies. We also present current research on the progress of developing novel routes of administration for ocular gene therapies. Retinal gene therapies offer the potential for life-changing treatment for chronic conditions like age-related macular degeneration with a single administration. In doing so, gene therapies change the landscape of treatment options for these chronic conditions for both patient and provider.

Original languageEnglish (US)
JournalEye (Basingstoke)
StateAccepted/In press - 2022

ASJC Scopus subject areas

  • Ophthalmology
  • Sensory Systems


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