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Gene Therapy in the Anterior Eye Segment.
Cynthia Amador1, Ruchi Shah1, Sean Ghiam2
1Eye Program, Board of Governors Regenerative Medicine Institute and Department of Biomedical Sciences, Cedars- Sinai Medical Center, Los Angeles, CA, USA.
Current Gene Therapy
|April 27, 2021
Summary
Gene therapy shows promise for anterior eye diseases, utilizing advanced delivery systems and gene editing. This review covers applications for conditions like corneal dystrophies and glaucoma, with ongoing research in human studies.
Area of Science:
- Ophthalmology
- Molecular Biology
- Genetics
Background:
- Gene therapy offers novel treatment strategies for anterior segment eye diseases.
- The ocular surface presents unique challenges and opportunities for gene therapy delivery.
Purpose of the Study:
- To comprehensively review gene therapy advances in the anterior eye segment.
- To analyze gene therapy applications for various ocular surface diseases and their outcomes.
Main Methods:
- Review of viral and non-viral gene delivery systems.
- Discussion of gene editing (CRISPR-Cas9) and epigenetic therapeutics (antisense, siRNA).
- Analysis of preclinical (in vitro, animal models) and clinical human studies.
Main Results:
- Gene therapy has been tested for corneal fibrosis, wound healing, graft survival, neovascularization, genetic dystrophies, herpetic keratitis, glaucoma, and dry eye.
- Promising outcomes observed in various disease models, with increasing human study data.
- CRISPR-Cas9 and siRNA therapeutics show significant potential.
Conclusions:
- Gene therapy is a rapidly expanding and promising field for treating diverse anterior segment eye diseases.
- Further research and clinical trials are essential to translate preclinical findings into effective human treatments.
- Advanced gene delivery and editing techniques are key to future ocular therapies.
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