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Retinoids for treatment of retinal diseases.

机译:维甲酸用于治疗视网膜疾病。

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Knowledge about retinal photoreceptor signal transduction and the visual cycle required for normal eyesight has increased exponentially over the past decade. Substantial progress in human genetics has facilitated the identification of candidate genes and complex networks underlying inherited retinal diseases. Natural mutations in animal models that mimic human diseases have been characterized and advanced genetic manipulation can now be used to generate small mammalian models of human retinal diseases. Pharmacological repair of defective visual processes in animal models not only validates their involvement in vision, but also provides great promise for the development of improved therapies for millions who are progressing towards blindness or are almost completely robbed of their eyesight.
机译:在过去十年中,有关视网膜感光受体信号转导和正常视力所需的视觉周期的知识呈指数增长。人类遗传学的实质性进展促进了候选基因的识别和遗传性视网膜疾病基础的复杂网络的识别。已经模拟了模仿人类疾病的动物模型中的自然突变,现在可以使用先进的基因操作来生成人类视网膜疾病的小型哺乳动物模型。对动物模型中有缺陷的视觉过程进行药理修复,不仅可以验证其参与视觉的能力,而且还为开发成千上万的失明患者或几乎完全丧失视力的治疗方法提供了广阔的前景。

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