视周期蛋白研究进展及临床翻译研究进展。

Xin Yee Ooi, Rujman Khan, Anjalee Choudhury, Francisco Xavier Elisarraras, Jeff Grigsby, Brandi Obregon, Andrew Tsin
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引用次数: 1

摘要

自我们上一篇论文《视觉周期蛋白:结构、功能和在人类视网膜疾病中的作用》(Tsin, et.al, JBC 293:13016, 2018)发表以来,本文在多个主题上取得了重大进展。在本文中,我们进一步探讨了DES1和IRBP两种视觉周期蛋白的研究进展。此外,我们强调其他视周期蛋白研究的临床转化进展,包括fda批准的Leber's先天性黑朦基因治疗的突破,以及各种视网膜疾病(如视网膜色素变性、糖尿病视网膜病变和Stargardt's病)临床试验不同阶段的额外基因治疗。
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Recent advances on visual cycle protein research and progress on clinical translation.

Since the publication of our previous paper, Visual cycle proteins: Structure, function, and roles in human retinal disease (Tsin, et.al, JBC 293:13016, 2018) there has been significant progress on multiple topics discussed in this paper. In the present communication, we further explore research advances on two visual cycle proteins: DES1 and IRBP. In addition, we emphasize the progress of clinical translation of other visual cycle protein research, including the breakthrough of FDA-approved gene therapy for Leber's congenital amaurosis, and additional gene therapies at different stages of clinical trials for various retinal diseases such as retinitis pigmentosa, diabetic retinopathy, and Stargardt's disease.

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