Can genome editing transform ocular disease treatment?

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Can genome editing transform ocular disease treatment? Nature UCIrvine ocular oculardisease eyedisease genetics genomeediting geneediting

By Pooja Toshniwal PahariaAug 3 2023Reviewed by Lily Ramsey, LLM In a recent review published in Experimental & Molecular Medicine, researchers reviewed existing data on the discovery and development of gene-editing technologies and their applications in managing ocular diseases.

In contrast, advancements in non-invasive imaging and electroretinography enable real-time safety and efficacy monitoring. CRISPR-Cas9 comprises single-guide ribonucleic acid , which targets a particular sequence of DNA and Cas9, and cuts the DNA molecule at the target location, giving rise to double-stranded breakages . The endogenous machinery uses either of the two mechanisms for break repair: homology-directed repair or non-homologous end joining .

In recent years, the fourth-generation CBE and phage-assisted non-continuous evolution , and continuous evolution methods have been applied to improve the efficiency and compatibility of the original ABEs. Applications of gene editing in managing ocular diseases The eye is an ideal candidate for gene therapy and genome-editing approaches due to its small size, immune privilege status, compartmentalization, and easy accessibility.

CRISPR-Cas9-mediated gene editing can potentially target the collagen type VIII alpha two chain mutation in the early-onset Fuchs endothelial corneal dystrophy mouse model, potentially circumventing the need for transplantation.

 

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