Review of Gene Editing for Treatment of Ocular Diseases
August 9, 2023| |
Researchers discussed the application of gene editing technologies to ocular diseases. Current limitations and ongoing research endeavors that will help overcome these issues were also reviewed.
An example of a successful ocular gene therapy is the voretigene neparvovec (Luxturna) which can be used to treat Leber congenital amaurosis (LCA), a disease caused by mutations in the RPE65 gene. Gene editing also played a major role in helping treat corneal diseases, glaucoma, pathologic neovascularization, and inherited retinal diseases.
The current limitations of gene editing include provoking immunogenic responses, which may compromise their efficacy and safety. There is also a persistent expression of genome editing tools, which may enhance the risk of off-target effects and unexpected consequences. To overcome these challenges, researchers should implement standardized procedures, thorough assessment of efficacy and safety, and comprehensive long-term follow-up studies.
For more information, read the journal article in Nature.
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