Galaxy Y Cho1,2, Yazeed Abdulla3, Jesse D Sengillo1,4, Sally Justus1,5, Kellie A Schaefer6, Alexander G Bassuk7, Stephen H Tsang1,2,5, Vinit B Mahajan8,9. 1. Edward S. Harkness Eye Institute, New York-Presbyterian Hospital, New York, NY, USA. 2. Department of Pathology & Cell Biology, Institute of Human Nutrition, College of Physicians and Surgeons, Columbia University, New York, NY, USA. 3. School of Medicine, The University of Jordan, Amman, Jordan. 4. State University of New York Downstate Medical Center, Brooklyn, NY, USA. 5. Jonas Children's Vision Care, and Bernard & Shirlee Brown Glaucoma Laboratory, Department of Ophthalmology, Columbia University Medical Center, New York, NY, USA. 6. Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, IA, USA. 7. Department of Pediatrics and Neurology, University of Iowa, Iowa City, IA, USA. 8. Omics Laboratory, Stanford University, Palo Alto, CA, USA. 9. Department of Ophthalmology, Byers Eye Institute, Stanford University, Palo Alto, CA, USA.
Abstract
PURPOSE OF REVIEW: Clustered regularly interspaced short palindromic repeats (CRISPR) is a genome engineering system with great potential for clinical applications due to its versatility and programmability. This review highlights the development and use of CRISPR-mediated ophthalmic genome surgery in recent years. RECENT FINDINGS: Diverse CRISPR techniques are in development to target a wide array of ophthalmic conditions, including inherited and acquired conditions. Preclinical disease modeling and recent successes in gene editing suggest potential efficacy of CRISPR as a therapeutic for inherited conditions. In particular, the treatment of Leber congenital amaurosis with CRISPR-mediated genome surgery is expected to reach clinical trials in the near future. SUMMARY: Treatment options for inherited retinal dystrophies are currently limited. CRISPR-mediated genome surgery methods may be able to address this unmet need in the future.
PURPOSE OF REVIEW: Clustered regularly interspaced short palindromic repeats (CRISPR) is a genome engineering system with great potential for clinical applications due to its versatility and programmability. This review highlights the development and use of CRISPR-mediated ophthalmic genome surgery in recent years. RECENT FINDINGS: Diverse CRISPR techniques are in development to target a wide array of ophthalmic conditions, including inherited and acquired conditions. Preclinical disease modeling and recent successes in gene editing suggest potential efficacy of CRISPR as a therapeutic for inherited conditions. In particular, the treatment of Leber congenital amaurosis with CRISPR-mediated genome surgery is expected to reach clinical trials in the near future. SUMMARY: Treatment options for inherited retinal dystrophies are currently limited. CRISPR-mediated genome surgery methods may be able to address this unmet need in the future.
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