Advancements in pre-clinical development of gene editing-based therapies to treat inherited retinal diseases

基于基因编辑的疗法在治疗遗传性视网膜疾病的临床前开发方面取得了进展

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Abstract

One of the major goals in the inherited retinal disease (IRD) field is to develop an effective therapy that can be applied to as many patients as possible. Significant progress has already been made toward this end, with gene editing at the forefront. The advancement of gene editing-based tools has been a recent focus of many research groups around the world. Here, we provide an update on the status of CRISPR/Cas-derived gene editors, promising options for delivery of these editing systems to the retina, and animal models that aid in pre-clinical testing of new IRD therapeutics.

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