基于基因编辑的治疗遗传性视网膜疾病的临床前开发进展。
Advancements in pre-clinical development of gene editing-based therapies to treat inherited retinal diseases.
机构信息
Division of Neuroscience, Oregon National Primate Research Center, Oregon Health and Science University, Beaverton, OR, United States.
Department of Ophthalmology, University of California San Francisco, CA, United States; Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research, University of California San Francisco, CA, United States.
出版信息
Vision Res. 2023 Aug;209:108257. doi: 10.1016/j.visres.2023.108257. Epub 2023 May 19.
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.
摘要
遗传性视网膜疾病（IRD）领域的主要目标之一是开发一种能够应用于尽可能多患者的有效疗法。为此，基因编辑已经取得了重大进展。基于基因编辑的工具的进步是世界各地许多研究小组最近的关注焦点。在这里，我们提供了 CRISPR/Cas 衍生基因编辑工具的最新状态，这些工具为将这些编辑系统递送到视网膜提供了有希望的选择，以及有助于新的 IRD 治疗药物临床前测试的动物模型。
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