Recent advances on visual cycle protein research and progress on clinical translation.

Archives of clinical and experimental ophthalmology(2020)

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摘要
Since the publication of our previous paper, (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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关键词
DES1,DR,Gene Therapy,IRBP,LCA,RP,RPE 65,STGD,Visual Cycle
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