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视网膜疾病小鼠模型的最新进展。

Current Advancements in Mouse Models of Retinal Disease.

机构信息

Department of Ophthalmology, University of Tennessee Health Sciences Center, Memphis, TN, USA.

Department of Medicine, University of Tennessee Health Sciences Center, Memphis, TN, USA.

出版信息

Adv Exp Med Biol. 2023;1415:371-376. doi: 10.1007/978-3-031-27681-1_54.

Abstract

The field of retinal degenerative (RDs) disease study has been in a state of exponential growth from discovering the underlying genetic components of such diseases as age-related macular degeneration (AMD) and retinitis pigmentosa (RP) to the first gene therapy developed and approved for human Leber congenital amaurosis. However, a source for high-fidelity animal models of these complex, multifactorial, and/or polygenic diseases is a need that has yet to be fulfilled. While models for AMD and RP do exist, they often require aging the animals for a year or more, feeding special diets, or introduction of external modulators such as exposure to cigarette smoke. Currently, work is being done to uncover high-fidelity naturally occurring models of these retinal diseases with the hope and intent of providing the vision community the tools it needs to better understand, treat, and, one day, cure the patients suffering from these devastating afflictions.

摘要

视网膜退行性疾病(RDs)的研究领域一直在呈指数级增长,从发现年龄相关性黄斑变性(AMD)和色素性视网膜炎(RP)等疾病的潜在遗传成分,到第一个为先天性黑蒙 Leber 患者开发并获得批准的基因治疗。然而,开发这些复杂、多因素和/或多基因疾病的高保真动物模型仍然是一个尚未满足的需求。虽然已经有 AMD 和 RP 的模型,但它们通常需要对动物进行一年或更长时间的老化,喂养特殊饮食,或引入外部调节剂,如暴露于香烟烟雾。目前,正在努力揭示这些视网膜疾病的高保真自然发生模型,希望并旨在为视觉科学界提供所需的工具,以更好地理解、治疗,并终有一天治愈这些毁灭性疾病的患者。

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