Clinical Implementation of Gene Supplementation and Genome CRISPR-surgery in RP
نویسندگان
چکیده
Purpose of review: Clustered regularly interspaced palindromic repeats (CRISPR) systems have 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 have been developed in order to target the wide array of ophthalmic conditions including inherited and acquired conditions. Gene rescue and disease model rescue in preclinical models (in vitro and in vivo) of ophthalmic conditions point towards the anticipated successes of CRISPR systems as therapeutics. In particular, treatment of Leber congenital amaurosis by CRISPR-mediated genome surgery is expected to be tested in clinical trials in the near future. Summary: Treatment options for inherited retinal dystrophies are currently limited. CRISPR-mediated genome surgery methods may soon be able to address this unmet need.
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