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Updated: Mar 8, 2026
Genome Editing in Mammalian Cell Lines using CRISPR-Cas
Published on: April 11, 2019
CRISPR applications in ophthalmologic genome surgery.
Thiago Cabral1, James E DiCarlo, Sally Justus
1aJonas Children's Vision Care, and Bernard & Shirlee Brown Glaucoma Laboratory, Department of Ophthalmology, Columbia University Medical Center bEdward S Harkness Eye Institute, New York-Presbyterian Hospital, New York, NY, USA cDepartment of Ophthalmology, Federal University of Espírito Santo, Vitoria, Brazil dDepartment of Ophthalmology, Federal University of São Paulo, São Paulo, Brazil eState University of New York Downstate Medical Center, Brooklyn, NY, USA fDepartment of Ophthalmology, Xin Hua Hospital Affiliated to Shanghai Jiao Tong University, Shanghai, China gDepartment of Pathology & Cell Biology, Institute of Human Nutrition, College of Physicians and Surgeons, Columbia University, New York, NY, USA.
Clustered regularly interspaced short palindromic repeats (CRISPR)-associated systems (Cas) show promise for genome editing in ophthalmology. This technology is advancing rapidly, with potential for clinical eye therapeutics soon.
Area of Science:
- Ophthalmology
- Genetics
- Molecular Biology
Background:
- Precision medicine utilizes genetic variability for disease treatment.
- CRISPR-Cas systems offer a novel approach to genome editing.
Purpose of the Study:
- To review and discuss the application of CRISPR-Cas systems for genome editing in ophthalmology.
Main Methods:
- Review of recent advancements in CRISPR-Cas technology for ocular applications.
- Analysis of preclinical models and in vitro studies using CRISPR-Cas for eye diseases.
Main Results:
- CRISPR-Cas has demonstrated progress in mammalian preclinical models of eye disease.
- In vivo modification of ocular tissue and stem cell correction have been achieved.
- Targeting of pathogenic alleles in vitro has been successfully performed.
Conclusions:
- CRISPR-Cas genome editing has corrected pathogenic mutations in vivo and in stem cells.
- While off-target effects are a concern, technological improvements are expected.
- CRISPR-Cas systems are likely to be used in clinical eye therapeutics in the near future.
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