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CRISPR-mediated Ophthalmic Genome Surgery. Current ophthalmology reports Cho, G. Y., Abdulla, Y. n., Sengillo, J. D., Justus, S. n., Schaefer, K. A., Bassuk, A. G., Tsang, S. H., Mahajan, V. B. 2017; 5 (3): 199–206

Abstract

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.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.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.

View details for PubMedID 28966884

View details for PubMedCentralID PMC5613978