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Recent progress in CRISPR/Cas9 system for eye disorders
D A Ayush Gowda1, Girish Birappa1, Sripriya Rajkumar1
1Graduate School of Biomedical Science and Engineering, Hanyang University, Seoul, South Korea.
Progress in Molecular Biology and Translational Science
|January 17, 2025
Summary
The CRISPR/Cas9 gene editing system shows promise for treating ocular disorders by correcting genetic mutations. This technology is advancing the development of new therapies for eye diseases.
Area of Science:
- Ophthalmology
- Genetics
- Molecular Biology
Background:
- Ocular disorders present diverse symptoms due to genetic and environmental factors.
- The eye's unique structure supports advanced gene editing therapies.
- Genome editing offers potential treatments for various diseases, including eye conditions.
Purpose of the Study:
- To explore the application of CRISPR/Cas9 technology in ophthalmology.
- To discuss the generation of ocular disease models using CRISPR/Cas9.
- To review current and future therapeutic uses of CRISPR/Cas9 for eye pathologies.
Main Methods:
- Utilizing the CRISPR/Cas9 system for gene editing in ocular research.
- Generating ocular disease models in mammalian cell lines and in vivo.
- Investigating the correction of mutations in patient-derived cells.
Main Results:
- CRISPR/Cas9 enables gene modification in mammalian models of eye development and disease.
- Successful correction of harmful mutations in patient-derived ocular cells.
- Development of various ocular disease models for further study.
Conclusions:
- CRISPR/Cas9 technology is a powerful tool for studying and potentially treating ocular disorders.
- Further research is needed to overcome challenges and fully realize therapeutic potential.
- This gene editing system holds significant promise for the future of ophthalmology.
