About this Research Topic
In the current era of personalized medicine, we have identified a large number of genetic variants in patients with various diseases using next generation sequencing. Recent advances in genetic engineering, genotyping, high-resolution imaging, and biomarker testing have made it easier to deliver the right treatments to the right patients at the right time. This article collection presents an overview of gene editing strategies including variants of CRISPR and Cas proteins, guide RNA (gRNA) selection and synthesis, gene editing, RNA editing, delivery to cell/tissue, and off-target analysis. These theoretical principles will be supplemented with the practical and clinical applications of genome engineering in eye and vision science.
Many basic- and clinician-scientists already have a general background of and appreciation for the CRISPR/Cas9 system due to its increasing popularity in scientific literature. This system, however, constitutes only one aspect of the larger field of gene editing. A deeper understanding of the technical and theoretical details of gene editing, in turn, may benefit researchers in designing and executing their own experiments. Given the rapid changes within this field, a comprehensive overview must invariably include a summary of recent developments and innovative techniques. As such, this collection will also address the updates that have emerged and their translational applications. To that end, we will describe how these cutting-edge techniques have facilitated new therapeutic avenues to ultimately treat retinal and other ophthalmic diseases.
Keywords: genetic engineering, gene editing, CRISPR, RNA editing, eye and vision science, retina, ophthalmic disease
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