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Vectors and Gene Delivery to the Retina.
Annual Review of Vision Science ( IF 5.0 ) Pub Date : 2017-09-25 , DOI: 10.1146/annurev-vision-102016-061413
Arthur Planul 1, 2, 3 , Deniz Dalkara 1, 2, 3
Affiliation  

One of the great advantages of the retina as a target tissue for gene delivery is the wide array of genetic tools that have been developed in the past decade. This includes a variety of vectors for therapeutic gene delivery to most types of retinal neurons and glia, as well as cell type-specific promoters for restricted gene expression in distinct neuronal subtypes. Within the scope of neuroscience applications and for gene therapy, it is now routine to express reporter genes, replacement genes, neuronal activity indicators, and microbial opsins in specific neuronal types in the mouse retina. However, there are considerable anatomical, physiological, immunological, and behavioral differences between the mouse and the human that limit the usefulness of these tools in humans and nonhuman primates. Several advances are now being made toward the goal of applying viral targeting tools to understand the primate retina. Here, we describe these advances, consider their potential to advance our understanding of the primate retina, and describe what will be needed to move forward.

中文翻译:

载体和基因向视网膜的传递。

视网膜作为基因传递的目标组织的一大优势是过去十年中开发出的各种各样的遗传工具。这包括用于将基因治疗性递送至大多数类型的视网膜神经元和神经胶质的多种载体,以及用于在不同的神经元亚型中限制基因表达的细胞类型特异性启动子。在神经科学应用和基因治疗的范围内,现在常规在小鼠视网膜中以特定神经元类型表达报道基因,替代基因,神经元活性指示剂和微生物视蛋白。但是,小鼠和人类之间在解剖,生理,免疫和行为上存在很大的差异,这限制了这些工具在人类和非人类灵长类动物中的有用性。现在正在朝着应用病毒靶向工具了解灵长类动物视网膜的目标取得一些进展。在这里,我们描述了这些进展,考虑了它们促进我们对灵长类视网膜的了解的潜力,并描述了前进的方向。
更新日期:2019-11-01
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