Abstract
In recent years, there has been accelerated growth of clustered regularly interspaced short palindromic repeats (CRISPR) genome surgery techniques. Genome surgery holds promise for diseases for which a cure currently does not exist. In the field of ophthalmology, CRISPR offers possibilities for treating inherited retinal dystrophies. The retina has little regenerative potential, which makes treatment particularly difficult. For such conditions, CRISPR genome surgery methods have shown great potential for therapeutic applications in animal models of retinal dystrophies. Much anticipation surrounds the potential for CRISPR as a therapeutic, as clinical trials of ophthalmic genome surgery are expected to begin as early as 2018. This mini-review summarizes preclinical CRISPR applications in the retina and current CRISPR clinical trials.
Highlights
After the first application of clustered regularly interspaced short palindromic repeats (CRISPR)-mediated gene-editing in human cells in 2013 (Cong et al, 2013; Jinek et al, 2013; Mali et al, 2013), clinical applications of the CRISPR system have become highly anticipated
Of pathogenic genes using CRISPR-Cas9, the pre-clinical in vivo models are outlined in this review because in vivo models point most directly toward potential clinical applications
The gene CEP290 is implicated in LCA10, which causes 30% of all Leber congenital amaurosis (LCA) (Maeder et al, 2015)
Summary
After the first application of clustered regularly interspaced short palindromic repeats (CRISPR)-mediated gene-editing in human cells in 2013 (Cong et al, 2013; Jinek et al, 2013; Mali et al, 2013), clinical applications of the CRISPR system have become highly anticipated. Control of or better understanding of off-targeting should be addressed before CRISPR can be implemented in a broader range of clinical applications. 10 CRISPR clinical trials that utilize the CRISPR genome editing tool are registered on ClinicalTrials.gov (Table 1).
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