Abstract
This chapter discusses the current status of therapeutic gene therapy strategies for the cornea. The cornea offers the ideal candidate for targeted gene therapy due to its small surface area, accessibility, ease of visualization, and its unique immune-privileged status. Corneal dystrophies are a group of inherited, largely monogenic, heterogeneous, bilateral disorders that affect the transparency or shape of the cornea. Clustered regularly interspaced short palindromic repeat (CRISPR)/Cas9 gene editing offers the opportunity to treat these disorders even before symptoms appear. Maximizing on-target cleavage while minimizing off-target cleavage is a major concern for the therapeutic use of CRISPR/Cas9, thus current efforts in the gene editing field are focused on optimizing these two parameters. In addition, delivery of CRISPR/Cas9 components to the cornea is a challenge, various viral and nonviral platforms are being explored to develop a vehicle that is highly efficient, specific, and nontoxic.
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