AOR
Archives of Ophthalmological Research

The Archives of Ophthalmological Research aims to publish issues related to publish articles of the highest scientific and clinical value at an international level, and accepts articles on these topics. The target audience of the journal included specialists and physicians working in ophthalmology, and other health professionals interested in these fields.

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Gene therapy applications in ophthalmology
Abstract
Gene therapy has gained significant progress in recent years. The success of these therapies is based on efficient gene transfer and stable transgene expression. With the US Food and Drug Administration’s (FDA) approval of gene therapy for Leber congenital amaurosis (LCA), the use of gene therapies has increased. Studies have provided new information about the benefits and limitations of using gene transfer routes to different regions of the eye. However, there are many old and new difficulties in the long-term effects, immunogenicity, targeting, and production of treatments. The eye’s susceptibility to inflammation and immune and inflammatory reactions encountered in gene therapy can render the treatment ineffective or harmful. Although many strategies have some requirements in common, it is not possible for a single gene therapy system to be used for all applications. Moreover, genome-engineering tools based on new delivery methods facilitate more effective and targeted applications to eye compartments. This review presents data on various approaches to gene therapy, their applications to various eye diseases from the anterior segment to the posterior segment, clinical limitations of current systems, future prospects, research aimed at alternative solutions, and advantages and disadvantages of gene transfer routes.

Keywords : Ocular diseases, Drug development, Ocular gene therapy, Gene editing, Gene therapy vectors

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Article available in :
Volume 2, Issue 4, 2025
Page : 69-74
https://doi.org/10.51271/AOR-0040
Article Information
Received : 06 Tem 2025
Accepted : 02 Ağu 2025
Published : 30 Eki 2025
Corresponding Author :

Kamuran Avcı: Department of Medical Genetic, Faculty of Medicine, Afyonkarahisar Health Science University, Afyonkarahisar, Turkiye

[email protected]
Citation : Eryiğit Eroğul L, Avcı K, Eroğlu Ö. Gene therapy applications in ophthalmology. Arch Ophthalmol Res. 2025;2(4):69-74.

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