Therapeutic applications of CRISPR/Cas9 gene editing technology for the treatment of ocular diseases

Abstract

Ocular diseases are a highly heterogeneous group of phenotypes, caused by a spectrum of genetic variants and environmental factors that exhibit diverse clinical symptoms. As a result of its anatomical location, structure and immune privilege, the eye is an ideal system to assess and validate novel genetic therapies. Advances in genome editing have revolutionized the field of biomedical science, enabling researchers to understand the biology behind disease mechanisms and allow the treatment of several health conditions, including ocular pathologies. The advent of clustered regularly interspaced short palindromic repeats (CRISPR)‐based gene editing facilitates efficient and specific genetic modifications in the nucleic acid sequence, resulting in permanent changes at the genomic level. This approach has advantages over other treatment strategies and is promising for the treatment of various genetic and non‐genetic ocular conditions. This review provides an overview of the CRISPR/CRISPR‐associated protein 9 (Cas9) system and summarizes recent advances in the therapeutic application of CRISPR/Cas9 for the treatment of various ocular pathologies, as well as future challenges.

Genome Editing VEGFA Prevents Corneal Neovascularization In Vivo

“…The CRISPR/Cas9 system has been used to study the role of specific genes at the cellular level and in living animals in the research of ophthalmic diseases. [ 25 ] For example, in a first phase I/II clinical trial approved by the United States Food and Drug Administration (FDA) in 2017, the CRISPR/Cas9 system was used to treat type 10 Leber congenital amaurosis (LCA), which is a congenital retinal dystrophy. In the past few decades, gene therapy has mainly been focused on small interfering RNA silencing VEGFA gene to treat neovascular eye disease.…”