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Advances in Gene Therapy for Retinal Diseases

A special issue of Journal of Clinical Medicine (ISSN 2077-0383). This special issue belongs to the section "Ophthalmology".

Deadline for manuscript submissions: 28 June 2026 | Viewed by 2093

Special Issue Editor


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Guest Editor
Department of Ophthalmology and Visual Science, University of Iowa Carver College of Medicine, 200 Hawkins Drive, Iowa City, IA 52242-1091, USA
Interests: gene therapy; neovascular age-related macular degeneration; diabetic retinopathy; central serous chorioretinopathy; metabolomics

Special Issue Information

Dear Colleagues,

The eye is considered an effective target for gene therapy due to its immune privileged status. Over the last few decades, there have been major strides made in the development of gene therapy for retinal diseases. In 2017, Luxturna was the first FDA-approved gene therapy in the United States for the treatment of Lebers Congenital Amaurosis Type 2. At present, there are several active gene therapy clinical trials for retinal conditions, including inherited retinal degenerations (IRDs), age-related macular degeneration and diabetic retinopathy. Gene therpies have shown promise in patients with IRDs where no alternate treatment options exist and in patients with neovascular age-related macular degeneration and diabetic retinopathy, where they present more durable and longer acting treatment options with the potential to decrease treatment burden and improve long-term clinical outcomes. Although gene therapy has the potential to revolutionize the way we treat patients with retinal diseases, we need to be mindful of the many challenges pertaining to drug safety/immunity, manufacturing/access and long term effects on ocular and systemic health.

This Special Issue aims to publish original articles that increase our undertanding of gene therapy in the treatment of retinal diseases. Submissions of review articles are also welcome.

Dr. Tahreem Aman Mir
Guest Editor

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 250 words) can be sent to the Editorial Office for assessment.

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Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2600 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

Keywords

  • gene therapy
  • inherited retinal degenerations
  • age-related macular degeneration
  • diabetic retinopathy

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Published Papers (1 paper)

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Review

21 pages, 3160 KB  
Review
Advances in Gene Therapy for Age-Related Macular Degeneration: A Narrative Review
by Noor-Us-Sabah Ahmad and Tahreem A. Mir
J. Clin. Med. 2026, 15(8), 3097; https://doi.org/10.3390/jcm15083097 - 18 Apr 2026
Viewed by 1689
Abstract
Age-related macular degeneration (AMD) is the most common cause of blindness and vision impairment in individuals over 60 years of age in the United States (US). Despite this, current treatment options have limitations related to drug efficacy and durability. Gene therapy provides a [...] Read more.
Age-related macular degeneration (AMD) is the most common cause of blindness and vision impairment in individuals over 60 years of age in the United States (US). Despite this, current treatment options have limitations related to drug efficacy and durability. Gene therapy provides a potential solution by providing a more durable and longer- acting treatment option that can decrease treatment burden and improve long-term visual outcomes. This review presents the current treatment approaches, routes of administration, and vectors being investigated for gene therapy delivery in AMD. It also provides an update on the ongoing gene therapy clinical trials for dry and wet AMD. As these therapies advance into later-stage clinical trials, ophthalmologists need to be mindful of the many challenges pertaining to gene therapy delivery, including safety, limitations related to immunogenicity, long-term ocular and systemic side effects, and potential barriers to drug manufacturing and access. Continued efforts are required to improve precision, safety, and efficacy, including identifying the safest and most effective vectors and delivery routes, and minimizing potential adverse effects. In addition, guidelines need to be established to guide appropriate patient selection before gene therapy can be integrated into clinical practice. Full article
(This article belongs to the Special Issue Advances in Gene Therapy for Retinal Diseases)
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